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2023-12-31-accounts

Chartty rnglstratlon numbBr1183996 MEDICS 4 RARE DISEASES ANNUAL REPORT AND UNAUDITED FINANCIAL STATEMENTS FOR THE YEAR ENDED 31 DECEMBER 2023

MEDICS 4 RARE DISEASES LEGAL AND ADMINISTRAYIVE INFORMATION TNsts83 Dr Debra Fha Mr Chrts France Dr OihAa GTÈnt MT DaNel J4ffries Mrs Undw Birrell 50¢rBt8ry DrLMRMcK8y Charlty numb¢r 1163996 Company nurnbor 1111È8 Prlnclpal 8ddresJ Unil 12 Troadaway T9chnI￿1 Centre Tre8daway Hill Lourtwater Hiah ￿t￿rnb HP10 9RS Bookkeepkng 4nd puyroll Adder BookkeeyrKJ Indvpendont Eyamlnor sa￿ttr ShahATII. FCA SKS Ramon LeB 93 Stsret LDfKIDn EC2A4BA

MEDICS 4 RARE DISEASES CONTENTS Pag TNstèas Annual feport Independent ey01n￿erfs report 11 Slafrmenl otffn8ndal adt¥ities 12 Balance sheel 13 Noles to Ihe fmand81 slolem8nls 14-24

MEDICS 4 RARE DISEASES TRIJSTEES. ANNUAL REPORr FOR THE YEAR ENDED 31 DECEMBER 2023 Th8 Tru5tse5 present thetr 8nnu81 ￿port And fln8ndal sl&lemenly farthe year ond8d 21 Dec8mber 2023. The financlal 518lemenls hav8 been prepared In 3(xx)rdanr Ihe 8ccounl'Thq PQtSdes sg1 oui lo nole I 10 the fiftantial Slatemen￿ 8r¥J u)mpW VAth tho Charit/s poveming doujmenl the ChaTiti85 2011 and ReporliTrg by Charttte5: St31ernenl ol R¢comnndèd Practi￿ appfThble lo thaTibes preparrng thetr octs)unts lft aLordancA wtth the Firan£Èa] R8pJrtin9 Stsndard 8ppfit>bl8 ￿ the UK and Repubfic ot Irelartd {FRS 102>" {effethive 1 January 2Q191. Bovomanco and monagoment Trnslees beEn apsKlnt8d io the M4RD boans based on thetr expertence. skiIls and enth(Esi8sm for the ol IA4RD. M4RD relle3 on the voluntsry Ibre Ih811ts Twsle88 [Jenerous￿ prDMde to gdvancg the pu¥p)5e5 QT the charity. Thg mlnlmLfO number of Ttuslees Is 3 8nd there 15 no m83Smum numbèr. The wttlrèments io be consldBr8d tsrthg W51Uon of TN51ee are oudlned In th8 Charfvs CIO Constibjtson. Twstte Indudonlg provkled by the CEO inpul fJTim thè Chalr ofTnJsle85, This15 CArriad out remDlety. Th& newTrustfra Is prfj￿ded ￿th B New Trusteo Induction Pack end awss to tho ChartV3 documents. M4RD makes ol read21y avallabl8 0￿{￿0 trBlnlng uealed by olker oryanisalions and a PDF txjpy of Thè ChArity Gorrnmityoll'5 doL3JThnl Yh? Essent181 True8'. Thg Ttusle Board me8ts quartety vfa vtdeo cothniung annuaty at a face-ll￿￿(e Strategy Moerfng. The CEO. Or LUGY Mckny. 15 8 fouThding mÉmber ol thg Gharity and has bgea in rule ol CEO swre September 2018. lJJc/ Is a key optnbn leader in Ihe rare dlseas8 tleld havlng had persDnal eyrienca tsf rare dlseasè and tralnlng In thé NHS 85 8 d¢Ktor. She reports lo the TnJstEo Board aftd Ès responsblg for. ￿nplementing th& str8teGy of tha ctharlty. as agreed by the Trustees; manaolrbg Ihe stsff team; ¢voTrlght ol tJnante3 and fundralsln& and IdenilfytrYJ and Bsses3irTrB strategic riskg Énd oppottLthities. The currènt M4RD staff team ￿￿P[l585. Jo MGPherscn Iopèrationg ènd F[na￿ MBnaget) tespoTr&bE8 for d8ly operalionj, managinG th& fltwnLE5. fundrd151ng 8Trd pauenl Adw group Ila150n: Dr Emma Huskinsort (Communlcations Lead) responsible for pubfJ¢ rn18tions, ￿aLsIng wilh press And writing med￿1 conlent: Ele8nuf ChurtiS1 (DSgital Prvlec15 Officer) resPon￿bIe for the development and delivery ol di9il81 pmJÈ¢ts. è,g. M4RD.. LEam: tha R8ts DLsth5e Podcasl 4 IAerfKs stri¢s: Bnd Ny￿1é Jefferies {Projed CoordinaiDr) responsible for del￿ring the MHS Éngland corntracl for Th& GeThr)mlcs Education p￿g￿￿e. itNolvttry the development of tho rare d{seg￿ hub and GeNoles. Luw, Jo and Emma WO￿ pall-timo. Brlh Ele8ncr ond N&tha fUl￿tiM0. Lucy and Nadine trom a hot dèsk In an office al tho buslness Analogva Wonderland. Hlgh Wcombe (ihe rtulsiered eddr¢55 of the Charftyl. A1 remeinln J sIAff w)ft from l)rfte Bnd report lo Lucy vrtw) tum ￿pOrtS lo th8 Chatr. Lw holds Y￿kly 1.1 rneetings th the 518ff team; Jo 8rd LUGY ￿￿et wBekty Ylth the Ch81r. Trg M4RD Board ofTruslees conlinue la cOntr￿Ule generous￿ to tho Sur)￿5 of the charfty. TvO Tnjslees are GPS And have been In M4RD (In rls prevlous fotmsl ￿nc8 2011. Dr Olivij H8nn8h Gr8nl Is gn Inlern8tson81 iacmsse plgyer, but for M4RD she provides Inslghl inlo dgy lo day Ime and learntng needs of belna 8 GP In the NHS. Tho Tre8yJTer. Dr Dèbr8 Finè. Vds an Ac£¢unlanl prfor to traln5ng to becorne 8 doclor and thls range ol 8￿￿nent4 Is Inrdl¥Jablo to th8 role. Al the Stralegy Nleetin9 Juty 2023 Lindsay BL￿ll Yras ￿ted in a5 ChBlr of M4RD. rep12dng Cmg France vth?se firsl tenn was complete. Und5 pIior experience sw>rkrAg for Melabafjc Support UK as CEO has been inv8lu2blo to Lucyand the resl of th& Tw$l¢e Board. Aftef ll vute by remaintng Trustees, Chris Fran￿ retumed lo> the Board for Ns Se￿nd tenTr as Tfu3toe. ch￿3. backoriyjnd Is In sB5es and h? found00 e suttssful wommerce company. Ho PeJ a pryotsl In getbng M4RD's structur¢ &gtRblishod, menforfng Lury and stèating tho thitity ui Vt0 (threcuon ofsuvxs5ful stsutegle& Now that M4RD has rnoy8d on from Its st8rt-up ph850 8nd h1$ to￿ had lo an end, Chris foll Itvray brn for & new Ch8ir lo be appolnted. Dan Jeffrfes 15 M4RD'J palienl rnpres¢ntalivg but h85 many S￿115 ￿thIn IT. HIS dayJob ol ¢tsaGng oniing inleradfvy educ8lron81 Soft%￿ moans he 15 able tp pro￿? bB5poka Ind grealty ￿sC￿nI&d seThlc•s In tho cTrBtion and ttovetopmenl of m4￿S tearing management 5ysiem {M4RD: Le8rn). Thg arn￿ 8nd obFLiMs oflne 2D23 plan fho d8ci&on m8kn9 duthg this

MEDICS 4 RARE DISEASES TRUStEES' ANNUAL REPORT (CONTINUED) FOR THE YEAR ENDED 31 DECEM8ER 2023 The Board ol TNstee8 8nd the CEO 8Tr re¥￿nsIb50 fff mrewqng and updaltng thB Ched￿$ strBleglc plan. Thls was rer17tW dona al M4RD's St{Ble￿Y Nloetsng tn Jdy 2023, othlining pf8ns lo d¢h£lop a QToNth S￿ling strategy forthe tharity. The CEO uses quatiefy BoaRI MeeYng5 lo widato Ihe Tw5[e￿ cn Fvogre53 being mad# by the and to ask quesbons and reoive ad¥Tee. The CEO is largety resptsns"Q)IB for dedsons about acJiwbes Ih8t achie the and objedlve￿ L8rgè fjnandal dedslons ILXrtsld8 of pr￿u￿0￿3ed prole¢ts) arv taken lo tho by the CEO th ba disojssed. objec￿V&S ind 8£llv11185 M4RD was reglthred at Companles House a5 a pdvale company reglslered by guBr8nl8e Wthoul sharB c8pltal (Coffg8ny Nurn￿r 11119884) on 201h De(¥mber 2017. It W8s rrgFStered 05 a tharity in t8nd & Walbs IReglsl¥ed Chgrfty Number I183￿6) on 20th JLJne 2019.11 bec4mo a Charifabl? Incorpor4ted Ofganlsallon ICIOI on 11 Ju￿2022. Below a￿ Iho chows purpoye5 as set out in Ihe ob5ed5 cfjnlained in tha Charivs CIO C¢nsttiutlon: cornpany is ¢staWislJed f¢r the obi¢rts of th4 r¢Sef ¢f sickness and p￿SerV1￿n of he8Kh 01 th05? %thruig from rafè dlsekses, thTouDhouithe VI￿d, by. (a) advandnu the edu(aUun ol mod1￿. ossodated ptofesslonals 8nd the wbllc In rnre dlse85es. genetlc and geocmle rnedidne (b) Frornoting rese8rth in atl are85 relating to r8re disEllS85. genetic Bnd gefjomic rnedicine ond publtslllnu Ihe usefvt resuits (c) promts￿ng knpro¥ed Care a¢trJ treatsnenl of thosè gjfferlng from rare dsseases. Aims and publ1¢ bene Tha Charity I￿rn$ to Imwoye the flyes of a certain POTtion ol the pubfic those liwing wth rare ￿l$eaSeS and their communittes. It dce5 Ihts Ihrough ralstng aw3ren¢ss of the rele¥all￿ of rdre dlscases In medical pradlce. Tha Ch8rfty p￿￿rte5 educ4tion dboui rnre thsease and opporttmili&s to develop a dwl(38n'5 ￿dE￿￿￿￿n9 of ljus la￿e pwlatlon group In thB UK In orderlo better 5eThe IL Revlow. DurfnG 2019 the Board Df Trustees ￿[ked vdth the Charity Commlsslon lo make Sure Ils s1Ju¢￿r0, obledg and go¥eThlNg d0￿MentS ￿adY for tha wBarusalion io b¢ KegistBTEd as 8 tharity. Thls Tequirod a re￿OW ol our p￿￿¢se5 tr) make Sure trty wer¢ Wiotty ¢eDtsbl8 2nd benèfittd publ1& In tho cas8 of M4RD the pubSd ￿fE to tha estimated 3.5 million people in the UK sjjfferftom a raTO dise8se- and their cornmuniuas. F¢Jltovang guidan￿ from the Chatity CvwwyT]155torE the eoonl of Trusiees adapted the compaws original Memorandum & NJlde5 lo make SU￿ the wjnling ws suitable for the tharfty. Subsequentjy the Charl￿S CIO Conslitsrtion Yffltten lo refted Lho Te(tmmcndabens from the Charity Commls&on. Th6 oruantsatiens obj2ds are vknolly charltsble. Any per5¢nal benefit arf&ng Is legmtmalety Irtddentsl. The ol TNstees rorfew M4RD's obJ8dives. otsals and straieoleg on &i artnual basis at the StrateDY Meeting. Tbls IK don6 vAth referenc8 io ouldence ccalAkned in lh6 Charity Commlsston's gtsldanr￿ on publ1¢ benefil.

MEDICS 4 RARE DISEASES TRUSTEES. ANNUAL REPORT (CONTINUED) FOR THE YEAR ENDED 31 DECEMBER 2023 ActlvlUgB'. A rar8 cfseaso is defil￿d ￿ lh8 UK as 8 rjtrnrfibon th£1 8ffeds feknpr than S In 20.000 peopie. 7,£h)O Trr8 ill38asg$ havo been idtrntiffed Ythith means thBI understandlng 8nd lea¢hlng aboul rare d15eases in merfic81 edLtsts)n Is 8XtreMe￿ challenging. Hovrtver this is a th8llenge that ￿ cannol lo 8vi)id beeoUSO appfoyinwtety 3.5 people In the UK ha￿ a rdre thseasp. So. eaL diseaso is uithYidualb rartr, 8re couectivety common. QI these dIseases, 80% are known to g8neltc ui origin 8ftd 70% p￿sent exdusivety In chlJdhtsC•J. HoYrtV8r, d@3plle thls laTge prevalence (equ￿&[ent tD all Bdulls In the UK suffer aslhmal. e8th IndJvidu8J ￿ndi￿On ffoets so fèw peopl8 th81 tAey ternl to be oveth)cked 8nd rrisundtrstood by he81th profBssionals. reseÈrthers, education 5gdal care protAder5 and th? generni pubfia Th18 IBck olAwareness AwN)ngsl ￿e￿ICal profes￿onSI5 P&TticuLgfy dovosl8bng &nd ran le8d lo extrEme di89noskn"c delgy. A porson vAth B rare dise850 wll wail on avernoa y&ar8 for an 8cx)Jrale dlagnosl They VAII see dDdors, rocelva mlsdlagnoses 8nd potenURlly even InÈppropri81è troGtrnent Tris larto Aftd Ardu)usJoumty to B diaonosis na$ been named Ihtr Dl8gnostL Otyssef. Thg th311eng25 ol hthg a dlse858 do not stop after gBiilng a tlleonosls. People I￿Ng cnrnplelety fliffETeftl Ise2s¢s olten report the ￿me diffi￿ltieS in terms of he81thcro. social CSf&, famfy Irf8. eflutation. s%rrk and ment8T health. There arv many wonderful d]arlle5 in th8 UK Ihal support rare dlsease patlents depen1f￿g on Ihelr dlsease or sFeLafic need, u￿b￿lla tharstres gJoxssful pubSc awartness carpaigrts. HoHever. M4RD speciffcally IArgets an eudlenca that nttbo￿ elsa has been SP&C4f• c¢nntrabng on o)uld mako the greatest ¢ the di8gnosbc odyssey and Iha th￿Qng6S thBtfollDw. m8dir£l sturttnls and dudotS. By tteatin9 8 medical PTofes51on equipped t0 5usp¢d. ￿￿gnose end m&n8ge rafo dis8Bse can help people Imno ￿1th rarB condSbons h8¥e Ihe best othts)me$ and tpath thetr fvu potenual. To do thls M4RD15 dtivlllg provlrtlng ed￿￿On and pTrdir41 tools for n￿dical s￿dentS end d(ots. ￿lhOUl 8PPTLipiiate traiftlng, the Ide8 that rar8 dlseases arfj Irrelerdnt to dinkol praGUc¢ continuo lo ￿￿trIbUt6 Ic4 Ihe dSaanosbc oty&sey. We remetty thlg by presenting the stattstC5 to our audlencf and 8skJng people liwng Wth rarg condillons to share ihelr slotltss. M4RD have developed a new apptDath lo saL8factsrty c•ver the 5ubleL# ol d15ease T%ilhtn ullderura¢uale 8nd posIGf8dll81e medie81 training. Fin8ty. wo're treating aur own rÈgourtes And promobng Oth&￿ ￿SoUrCeS ￿ order ID support medical professEon8ls ￿en they need help on the subjeclof rare dlse8se. M4RO p￿￿rIeS edu¢atton onltne evenis. &leamlng. sorJal medla. via p£dcastnd in per5ontrainlng events. WIdErne￿Ork. M4RD ￿[13b0T8t2S with mAny umbi8llA And dJseas&specific rar6 diseASe adwLy gTOLfPS In ordor to 8th18ve Its objects. To name a fevr. Cambrfdge Rare Dlsease 14Bh￿rk, GEnttltt Affjanc UK. Rarè Revotullrbn Klagazlne. BTvaktng Oown Barriers, Rare Community Nehwjrt Rorcm%nds. Motabofjc Support UK and EDIR M4RD also partners wth BÈactsn to njn tha Aftnual 'Studenl Vaira Piize. 055ay ￿MperI[1D￿ and ilh Th& Royal So(aety of Medidne to host its annual wwstum 7he Unusual Suspects.. Boih IJK rellIste￿d thariyes. M4RD bono support Imm ihe hea11h communical￿n$ agenryi errbJtivB. Th6 charity also depends on volunteeTS to conthijuta Its PmAetXs and speak 81 events. NI4RD was atso a I(WjF￿x￿9 organisalton of an h￿Or￿71 group caued Action lor Rare Dise2$0 ET￿￿tThent (ARDEnt). 8kinggld8 Cambrfdge Raffj Olseases Net￿￿1￿ and Rale Revolutjon Magazln In 2023 M4RD expanded Its 0)r￿ectsDnS vdlhin nwdloai edurAtion to W0￿1n￿ wlh Th8 G8nonrfa4 Educat(¢n P￿gramm8 in earty 2023 to update &yp8nd g)nlent on IE5 Ram Dlsease Educabon Hub ￿ebpa99. M4RD 8rn llow+MJrtIrng lo prnvtdg othor I￿1¢nI for tho ￿b5{19 dnd for GoNulD$. Alongsldo RDIG Wa1è5, uD1ve￿Ity ol Cardlff and The Unlv8rsdy ol O>fofd. M4RD subrnlttvd A bld for lJndino by Lifath trjr 8 Tran51atitsnBI Rgseorch Centsv for Rarg Di5ga50. M4RD conbtsulod to deyi9ning a new scrvico for sse5StrvJ and suppurtsno pèopl8 rnrè corsditlonj In k￿lIh. sodal carn. oducatlDn ond ernployTront bia a hyblld dtgttal gnd la￿t￿laCe enhanced pack80è of care. ￿?￿y)rt from one vl ils Partnw5. M4RD h85 b8en thg coi4>ry MwfjcsAwlpry to uoat• Raro DSs&as8 101 tsalning for Feurd4lh)n Pro3r4mmp d¢xlor&

MEDICS 4 RARE DISEASES TRUSTEES. ANNUAL REPORT (CONTINUED) FOR THE YEAR ENDED 31 DECEMBER 2023 M4RD collaborated Medscape lo Inlerpr8t end publSsh a study ol th6 gwareness and leamlng needs of neaty IOOD dwuaans in the US and I¥￿8%￿&5) EU5. Dèclarntlon of Paymbftt to Thistsè In 2023 Dan Jeffrfes was paid £900.00 h Iclal to fvJrthet develop WRD'S 128mlng managEmenl gy3tern. 'KI4RD: Lèam. and to (feala an adtfitional Ment81 Health module. Pas7nenls to Tmsiees for Serw￿5 8re auowed by th8 charitys go¥e[n￿￿ do(3MnBnt PrD￿ded ￿ndItIOnS In su￿9U$¢ 62 are satisff ?d. Thoso condlUon2 S81isfled and th¢ Board ol TnJsie¢s IFJxdU￿ng Jeffrf?s} decided that hlring Dan Jeffries for trus specifjr sor¥ica in the be3t lnIe￿st af thg chadw glvun hls In depth kn¢JvAedge of the th8rity. hls pers¢nAI ¢xperfen¢B of IlvSnu ￿th iwv r8fg ¢isea599 and his prOfe￿ort￿ experi￿c in thTrg &ie8. AchluvemontB and porfonnance Chalr comments 2023 hBS been a ye8r of refiection on the thartvs PWTess so f8r, frDm ¢Vs slaE1 up rooig vthero a small team look an Id88 In 2018 and lu the lesL IhrDugh ts) now ￿Ing cons1de￿I by &akeholders suth as PAGS. educalknn eWishmenls. D¢paitth0nt of Ho￿th and Sodal ca￿ 95 being 8n Important voice thal cAn help sh8pa and deliver educatlan Gnd s4DpJrt to Hedihcwe Profes5i0s]als (HPCsl. therefore trnpmythg otrtcomÈs for th058 ￿n9 wFth Trrg conditions. In 8 ne￿￿ of hLtndrnds of patient #d￿raLY grwps and tr8ilbl￿nO r8w Otse8se 4)e¢fi¢ thaTities, M4RD has c¥eated 8 SP8e8 ta unlquely 8dvoc81è lor an innovauve waylo defiver eduGlt5on lo HCFS tmprovtng the ￿ d]se8se experfenco f¢r 811 patients regardless of Iheir 5peciffG wnditon. The nobon IW HCPS c4nnot po￿l￿1Y know how lo Identify and rn8n8gg Èll 7000 spedfiG rdre dlseBS85 has driven the e¥Dlutton of Idea thal by ryeallng and defNerfno education that Flaces ￿rtaIn unth8nBlng prfncipits Al the heartof HCP praclice th8y ran ba bettor equipped lo support those with rare disoases. Throuoh its key AthiEes of ofttirtè educstiM, and 8 fl4wiishinu Proufammo. M4RD has bpuun helplng HCPS ba more effecltve In a way that allow kn&iwdualy 8 rare cx)nditTon lo be Identsfied. nosed. a££%ssthe rigTrrtheallhwe and w>poTL Engaa8 systems and have bctter oUtc￿me5. Tha fnjsteès 8nd stAff lesm befieva In th6 Msion fflorn than èyer seen this pn)of of concept pmduce ev1den￿ and data lo ￿ve o)nfjdenco th811ts he￿ng an Impacivthero it ¥vas trdended. We belleve thatwth a new 5tsalegy In 2024, focusstrd re50lI￿es. uwvAh tsl InGome end Ihg passlon and èypett'se of Iho Icam, tho chBnty can GrDW In the sector Bnd be a key dettbtrer of edueayon helplng to qeate a ntw wave ol HGPS arv better supported provh¥a CBre forthose liwnp Kith r8re cond￿1)n$. We looked 81 th8 thaws p￿￿1￿$3 and fvtuffj dimthun thg cont8x1 cl tho thanggs 8nd thatlenue31ft pobcy hea1￿C￿r￿ Ènthnments in 5th11* ￿ operate kn Ehè UK 8lld rJJrnmiileiJ to ue8tlrg ¥olutions based on muiil akehtslder perspedives suth AS PAtlents. HCPS, pobcy makers ond 8dLtCBlioTh pmvlders. In 8ddition the thartty h8s exlended 115 glob81 gknjg us earty Inth¢2trM that this epproach to ed￿￿￿0Th o)uld b? lakBn ￿rfdfftde. The Bosrd ol Twstees would ￿k2 to thank Lucy. Jo, Narfne. Eteanor and Emma lor ernbr8c￿g this year of challenge and reffecdon. They have been ultiral In developlng a ambllkjus strategy thlth ￿1[ be launched In 2024. Wthoul Ihetr open nzndedn¢5S and Ihls new 5tsulw yitlh 115 tremendous potential ￿Uld nct hava been pos51ble. l am exdtod 81)oul th8 ygArshead. My petson31 Fughllghts frnm 2023 Indudts Tho Unugjd Suspecas 8t The Roy21 Sodèty ol me￿cl￿O gaw In51sht Into how M4FID5 0￿1n8 edu￿￿On bas Irrproved HCP rarB and p8lenl expgrfence In Breas su¢h as mental heakn.11 dso dèmonstralÈd how Ihmuoh thA arob•th ol iLqknbawdor Pfowamme rncre wofe5slonaly are y￿rking in 4 n8wway to Improv9 Outtsrnes f¢r thosa wth rate diseasas.

MEDICS 4 RARE DISEASES TRUSTEES. ANFIUAL REPORT (CONTINUED) FOR THE YEAR ENDED 31 DECEM8ER 2023 The maln olms ol 2023 Host 10th annual sympositrn tho The R￿81 SDdety Df Methiyte Urnisual Susped5: r8 dise8se in evgryday med4cino'. achlgved on 15th Febwary. Run M4ROsfi￿I sltuolured w8Trness c£mpa&n for Rare Disease Day on 28th FebNary- ctmpleled. . Expand orslne leomiftu pltforrn. M4RD: LEa71. Mental HEÈtih and Rare Dise#so launthed in 20rJ. ColJabornl8 varh the Rare DI￿8$9 NeN¥ort lo author, desfgn uealg and publish a nurslnu specific verslon of Rarn Di5eas¢ 101 to be hosted on M4FID.. Leam plarform - Due 19 evpnts outskle of WRD'S control w8 havè unfortt[nate￿ not béen able to ￿MPl￿1￿ thls proled 8nd funds wero dit8ded kn10 8 blggor menlal he8llh t>wrs8 Instead.As we now a sped&Ysl on Iha knb83S8dor Pn)gr8mmg. be takrno t￿$ prolect Into 2024 vnthg 8lon9&de the Global Rare Diseas8 Nurso U￿￿8￿radUate edu¢xbon: ts)nlinuè eMversations wilh Meditsl SLools a￿ul Rarg Disease 101 edueAtton - completed and onaolng. Pos1gradL￿e Irwning: ccnlnue ptDwthg Rare D￿ase 101 trthing tD doc£ors, ￿ped￿JY bTthh GP. Paediatiics and Emeruency Merfiunè- ts)mpleted and cngoing. Cre8t8 'R8re Dise8s8 In Prim8ry Cafv'_ rx Tnodel guidetine- Dr H3nn8h Ctsnl researthed and Mrnle 8 Rare DlseasD summary tor Piiroary Care has had Intgrest from a famous Prlmary Care edurzllon provlder for pubUc4tion as a 'Red 1fv18 P¢Arf. Tljasè a￿ fm ￿5M￿n rB50JJn%s Ihat 8rn shared eath ￿ek coveflng k8y prfmary car8 ioples. Coued data on sludenl under5tandSw Bb¢)ut raro dlsease and thelr leamlng needy ￿ tho UK RISE PflThe UK Rarn Diseasas Fonm t￿￿￿￿1￿9 the Qualty Stsndeids Independent￿thIS0ry Group. Thè Unusualsuspects: Rare dlsoase In overyday mfrdlclno 2023 The o¥enl th 2023 f¢oJssed on mental welTr•elng hi rare ￿lSease5 End WAS held in a ￿rid lormal this In 8SsodaYonWth the M¢dlEd Gene￿¢5 Seutlon oltha Royal Sodety of Metjidne. 278 partidp2nls reglstered far the event vAth 55 people attending In pefson and 103]olnlng us online. The recording has also been vRlthed oNlemurKI by fhose btho couldn't make itun the dgy. Alull report from the ovenl Is a￿17able here. Before tlu$ meeting, tsnly 43% vl part5ripanls Y￿re aware thal 3.5 MTI ion Ftopte in the UK (1 in 171 Bre Bffeded by a Dre dise&￿ In Lhtir litetiThl 'Il ￿an9￿d mypurc8ption on Ta￿ di￿￿05 and&auallYfvrth&r￿ myinter85fJn 1£ hoping that I CruldWal￿ti i 'As a mgdical Stt￿en1 I found Iho Kebin&r very ingohthil. ￿ hÉv8 limited tsaowng on rnrg rfw8S64 ￿ l ￿)uld mm+￿d8(10nding futura ￿blowS. tD mype9￿. fvlany 1hRn￿% to th& RSM Mèdlcal G8n8tL8 Sèdlon for SUPPCsrtlno u wilh our evÈnl ond gllovlnp us to usa thalr pres1Kd￿us plaNornL

MEDICS 4 RARE DISEASES TRUSTEES'ANNUAL REPORT (CONTINUED) FOR THE YE4R ENDED 31 DECEMBER 2023 Rare Dlseasg Day M414D's ¥ ShoYlrfowSlrip8S for # RareDiseaseDty canpwgn 2023 wds M4RD's fvsl ehEr R8¥0 Dise1$8 Day campalon. Our message was llGhthe2rted and str81ohl fot%Yard. targettng healthc8rn professionals: askln9 thern lo show their support ror people Mryth raro conditions by Mearing stiipgy 50(¥ for RaTr Dioease Dayl W8 a hug9 mwkout of eY￿1￿[T[g. shlny ted, b[and￿ eA￿lope5 (that could not ova1ookedl) atld posled io key tstgets. We incJuded iebrd stiipe sod(s 10gelher 8 branded Fin. and leaftets and poster5 vAth tsur key rnessbges. We Fan a fulj sodal rr￿8 rAmpdgn alofjgstdtt. 10 4JTiVB ihe me55aglng and makntain momentum In ths weeks leadtng up to the big day. Our • ShthYYoLrfttipes gallery Is a heartening demonstration Qf how 18r And knidB Iha mess8u8 rEathod. In lot4173 parc¢ls ￿re ￿nI. and 342 peopfo got InvDlved th8 c8rnpalpn. 203 phoios wern shared whid) èllhet IAaued us @medICs4re￿L3ea￿5 or used th8 ha￿g 4 shDwurstsfpBs. Some notablè larg8 Accounts shared thelr psrtEdpat(on 8.0. Sardh JaThrfs 36.K lollowars. Hor # shthYYou￿lpeS post 98ined 287 ￿ke$ 8nd 43 r&knEeL%. B8T¢rèsy Nicola B18GkKoL¥J shared a plclure of her so¢k5 on show In Tha House of Lords. to her ac(yJunl of 17.2K foIEo%rs. Genomtrs Endafjd. the BIU 5oulh8mpton and Camtsldg& UniveN-ty Ho4Jil&ls and Queen Mary Unlversity of London also I0￿ed 5n! The c8mpatgn ￿athed multiple countries emund the ￿rtd. induding tha US. ￿4]51[arIa end even Kaz&kP&stan. M4RD.. Learn The new onlSne wurse, 'Ment&l Haelfh End R8r8 Dlse8S8' was l$un<ed In thjly In collBbomlon wth RBremlnds, slsltng ¢f 8 Inlertth le5son5 e8th fi>2[￿9 Dn a Merenl toplc related to thg thallenges Ih81 livtrtu y￿th u raye dsease poses to a petsons menlal he8th, This course part of M4RD: Le8m p181fomi gn¢J o)mglements M4FiD'g exlsthig 'R8te Dlse889 101, ￿llr9e. k ￿1th all ol M4RD's leBming ￿ntenL thts course has been de1￿￿ed vlth peupla with rea￿kn￿d èwrienc At tha csJts. Le5S0fi5 indud? novel S￿ne￿o5 spellific dwllenBes suth as Ihe presence of Én Inheritsble or genefjc rxindibrffi Énd the bnpad ft M85 Qn palfents 8nd 18m11￿$. Th2Tr 8re pr8clic81 tips on how lo bBtter cJrnmunlcale £boLrt mental heaTth, wilh p8tients and carets.11 also I[K4u￿8 slEPS that medics can io thetr omn merrtal health y￿￿$t wppoitng the mentsl hearth ol others. Thv course has 127 partic3pants and bas ￿Uch1 thè #tlention of staXeholdef5 as a first 8ttempl to de￿er thts thd oftralnha. 4R As ol the end of 2023, has 1000 users and lls rn81erlal 15 b￿r0 used lo Inform re50UtC£5 for other healihL8re edvLzlor M4RDAmbasgadorn Ptooftmma In JLty. f2 ambassadors attendèd our fjrst over F2F meefna In Londonl P8Vellt 8dvo¢8les. medlcal students artd (thidan5 flljm 80 O￿T tho UK. c8ma together to celebFats thè end ol thelr fl￿ ar2deMl¢ year on the pwogrnmr and lo share $1¢ri63 and experfenres. engaglng and Insplrfng eBch other. 7he ambassadctrdayreafy swds èeffer than l eVer￿￿eded. Ileft leèling fike I had a new sEnsa ofpu and Importance lo cary the olhLJS ol M4RD in my din1￿1 Kork 8s a medi¢al thdenf and then gs 8 doclor. I really en￿y&l all the spewe5 hearing tho ambassadv￿ &scuss their I￿￿0Ver￿9 past ypar It Y￿$ weBt to hear frnm 8yeryong1- In SeplEfflberwa [c￿Med 25 new amb8s5adws to Iho programm8 and M4RD nuwhas 39 ambassadors madfr up df 16 rfinid8ns. 12 medic41 sludtnls and 11 patient lldvocales. Thls yoar v￿lCOme￿ an Maotholisl. Speerth & L8nBu8a8 Theraptst. Geneucjsl and Gllnlcal Nufse Spodaust Dnic IhB cfinScal programma. and our knqeosed numbèrolmedifsl sthdènts enabl85 us to awss rne&ic&l sd)ool$ Ulc Dwinu 2023. follo￿7￿9 ¢))TMplelion of Trd(n Tng Trainer. our rn&Siczl stud2nt and ￿niVAl amb4ssadth3 delivBfBd R2[9 101 traintno b) rnoTr than 6 settings 8fJDSS th8 UIL Jo rtms Iho amba55ador pnjgwnme. Ma￿lainInG ￿gUIar ¢Ontad thtvUgl￿ut the 3cad8rntc year with onfme Tevl se$570n5. TJ3M TreT￿nett sesston4 an ts￿1ne fonxm on M4RD LeAm 80d a ￿8t&4pp aroup. bAssada The M4RD vnuld fjko t¢ thank al Iha ￿ for plafva gjrh a Pl￿1￿ rolo in en8bfmg M4RD lo ￿[k th tha knlder dlsease crJmMUn￿ and nthing suro thal the PAflenl volce Is hgaKIl Ihniugh ow

MEDICS 4 RARE DISEASES TRUSTEES. ANNUAL REPORT (CONTINUED) FOR THE YEAR ENDED 31 DECEMBER 2023 Modlcal s￿dent placements M4RD look on Luttie Mercer, a 4th yeer Thedkal studenl al The Un5versty of Soulhamplon. In April tor 2 ¥hEeks. Du¥iTrg this tiD)9 Lottie rEYewod our Mentsi Fle81th Modula, End spoke knilh Sarsh V%fvi, CEO of Unique, and Angela Corrrwall. fouN¥er ol Diamond Eiacknn Anaemia. In Lottle"s bbg. one ol the tnairk she leami was the posr ol fistening. fiDm Lotye." A bi? thankycu fo ￿rfRD. ha¥e Il￿MedM? onfu th￿r(earn lor the past CLiup16 01 ￿kS As part ol my final ￿￿r eledlvg pla￿￿￿nt. It giè81 lo hatr¥ opwrttrrjily to mèel peopb frnm somg of ihg pationt adv(￿¢y gDups th6y wth, lo h88r abotst their puptsrignGes 8nd inlprndion5 healthta p￿re&s￿￿91$. 8s ¥%vII BS gJnlng gXP)SUTr to PU￿1¢ 8}yw￿ 8nd dis$8se wlry making. Qudng pla￿Ment, 181so began b r8co9nff￿ 8nLI appropale lh9 o)mplgx interplèy botb￿&ll mertlal and physlc31 h&8lth, 8ndieffeL¥ up)4 howI￿n support the menlalh8alth ofp90pIg liviw svlh rare dis88ses onc¥ I start 8S 8 junkjr Undorgraduata Rar8 Dlsea80 101 TralnFng Underyrdduale tratniThg events for medical slurjents held al Cambrfdge. Barts & The London. Dundee. 8sgow UnNerglÈs. LUGY dso spoke at th&AJ)nu81 Stydents for Global Health Confèrénco, Poslgraduats Rare Dlsaase 101 Trdlnlng 6 GP TraJnlng events held al￿35 England and Wal&a. 3 Fourh18ttonYe8rTraiNng e4tnlS In Entslan 7 PaethatrtcTr8lnlng Events. 1 Ph8nnaGy Trnin>nu Evenl. M4RD had an ext￿bill0n tsb￿ at the East Genomics Merfi(¥ne ShovK8se speaktnq lo postBraduales within the NHS. UK Rare Dlyeaseg Framowork M4RD ￿ntEnUeS io be repreSen￿d by Lucy on Ih8 LIK Diseases Frame￿￿[k Fowm vknith provide8 Q￿ertIs& to thè UK Implementation Group Ènd Thè Deparkneftl ol Heath and Swaal Ca￿. Lucy has been Y￿r￿9 a Dff.shool of this grJup that is y￿)[kInG NIGE to design Qualtty StsniJ8td5 for raie disea50 fot the lirne. It Is tharths lo this Forurn th81 M4RD has been c¢lt?borabng The Genorrdcs Edu￿[[￿ Programm¢ (p2rt of NHS Enyland) to ueale rare dls2ase resoyjrces for the websila and for GeNotes. GeNoles PTovides quir, c£nds 1¥rfoWrt￿tiOTr to he￿ healthcaru pmle&sion8ts make tho rfght genDrniC dEd￿onS Ét eath slaga of a dinlc£l Path￿. Meclies4RargDlse85e5 Yfds rrenuoned In the Enuland Artton Plan for R2Jr Dlseases publlshed on Rar6 DLseasa Day 2023, The Rarn DlsoasB Podcast 4 Plodlrs By the end ot 2023 the pod￿st had hll 8699 do￿lOadS and 48 eplsedes of the 5)odcast lavtr been publlshed. Season 5's episode.'ALK Positrve L￿a Csnrr 8rtd M8. vdtth Debra lknntsgue. has had 163 dDV+nhJads and is so fBr ourstdh rTr)slli3lened to podcast eyer. 'Frnm PlelbJ)ume to Manthester- a dinlr21 trials Sto￿ Tectived 135 do￿10&d% In the first 7 days making tt into thg fop 25% ofpodc4s1 episodes on PAJZZSPTC Tolal ulobal down]oad ￿l$1￿￿UlE0￿." 5,755 d￿￿[oadS In EurDPO. 2238 downl¢ads In NvrthAmgrfca. 318 dr￿￿0#￿$ In Asla. 44 InAJrica, 321 In O￿a￿a and 21 doymloads in SouthArneiic4.

MEDICS4 RARE DISEASES TRUSTEES'ANNUAL REPORT (CONTINUED) FOR THE YEAR ENDED 31 DECEMBER 2023 The StudÈnl Volto Prfie 2023 M4RD In a 50.50 partherslup with Beottjn lo produce th9 Student Volr Pf6ze. FoTrowing thé suceess of the 2022 S￿. M4RD end Beacon cothosted an online student event on Roro Oisease Day 2W23'Beyond the SbJdent Votce Ptizo. . Th8 trLerne of Ihe EvenIng was to IbghVght tho vrfec raTrg& of opporturntios ÈvaD8ble Ituvugh a ca￿er fogjsed on rare thsesse 8Nd the exciting luther opportunities a¥ali able from inifN>duals InVD1￿ In varfous aspeds of r8rè dls88se. The eyent was attended by 28 p8rtldp8nts. In Odober 2023 thg SVP launthed lof it3 101h yoar, spctnsoffjd by tho global medie81 coivnunlcatsons company EmoiNo. Thts quBsUons fcw5sed on Corn￿￿[U￿1JQll and Induswty In rdrn di5eBSB. Wè ￿tived foo ertsies and pal￿d 69 of theso sludents with pauenls throuuh p8lwfng gd)eme. Engllsh vRs not the natfve language for 27 of thè thdenls submltted ess8yS. Eswg submitted from 18 dtfforent ￿un￿fe8. In compartson to 2022 thera vrns 9 inue8se tft thB nunther uf essays enlet8d and a 45% b]ryease sn t￿y5 sJbsn(tted by studenis look part In the paU?nt P8lrtno scth8m8. RISE UKsuNey We 8ro in tho process 01￿[ling up 8nd submitring the re￿￿15 from The UK RISE Swvffy eXet￿d In 4))IlaboraUoTh ¥￿th Dr Dtmcan Ccle at of Carditl. We had 257 responses from 32 mediLI sthools 811 ov8rth8 UK {8114 natsons). Re5utts deatly $￿W theffj Is for lrnprnvefflent in rare dlsease educ4tion ￿thin medM?I und8ror2du8le progr2mg. Some ol the ￿publiShed flnjfings show Ihal lh¢ ThalDrity Dr respondents rnled thElr undofSt2ft￿lng of rnre dlseBses as la￿ end were +th5s8bsffod Hith th8 exlenl ef r8fB dTseas9 ediL8tson they had re￿￿e￿, ThIs WB8 reflected In thetr a￿[￿nesS of rgre rfjseases and thelr vnderstsnding of the pr&valÈnc and Impact of ih8sB cxjndiuons vathoi the UK. Wa look forwanJ to pubb8hing the PBper tu 8 medical IDiKn81 lor peor 9wln 2024. Fln8n¢lal Posttlon The budget for thls finanrlal yearwds dedded based on thè g0415 sei out to B(18￿ In 2023 wh5¢h rollecl the RD stretegy. We sel out lo fvndratse E247k orfer b fvnd 8diMties for 2023. In total £214k of fundin9 was se￿[8d throush spon50rshlp and d¢nalions. TOtaf￿ndS r2nSed fctW3rd L8 £92.995.The fing1 c8th posilon Is £79.270. £11.000 olvthith Is kept in o saving3 acDJunt as per th8 thBritys Reserves Policy. Prfnelpal Fundlno Sources The pthdp81 tunding 50Ufces for trus pertLYJ ￿rts sponsorshlp and d¢natfons from ￿MMerda[ companles. h)du￿Tru phaftna¢utir41 cornp8ni8s. Thts5e Meru.. AJexton Ther8peUti￿. Wlam, Amicu5 TherBpeuti£s, Blocrysl. Blonkél Emas. Chlesl, GDead. Kyobwd Ktrfn. Orthard Thernp8Utius, Wtc Ther4peuUGg, Sanoll, SOBI. Takeda UK and UC8. Charflable grnnls ￿re also received from tho Kemeth Hkroresves Tntsl RKf ChaTtlabl8 T￿￿1. thè FoThester Fanwty Trust end Scut?r Chatflabfe TNst. Resoryes Pollcy imd Rgvlew Th¢ Re5eNes Poliw VR5 put tn pl8(¥ by The Boanl ul Tnjstees in 2019. ReseNes are to be malntstned at a level l( ¢NgJies thai the tharivs ojm 8thMty could tsnbnue during 8 3 month peii¢Jd Tjf unfoteseen finar￿al thfficulty durfng fundlng Is lo b8 seujred. If Tundmg Is not seatred then thls Is by a 3 month period In Mlili th2 ora8nisatiDn cijuld be thssolved ￿T(h vIf debi setU8t1. Th8 Board otTrustaes havo sat a Min￿￿rn of £11,ODO to b& held in the re8erv&3. Flnanclal Rlsk The Etaff team and Tluslees (7n all Irom horne or NnvteTy In an apprnpri81o Ioc4tion so Ihe chadty doesnl own propety or hawg 8 long t8mi rental contratt. M4RD's blGgest llnandal ¢xmmltrnenl Is stsff. malgrfty ul the c8riVs knork is dona fft house. ￿ pmvided to M4RD on a pro bono basls thèrefDre exlemBI contr8tts for semces ar8 k•plto a Thllnlmu￿. M4RD tsysbtwas ta bank vitth CAF BBnk Mthlth all￿ 8 owdlgvel offjnanoai overslohlby Iho T￿a5￿￿ 8nd Dlher Trustees. All the Trustees have to Ihe LMlne bankjng system and al paymerts reqwre 2￿erSOn

MEDICS 4 RARE DISEASES TRUSTEES'ANNUAL REPORT (CONTINUÉD) FOR THE YEAR ENDED 31 DECEMBER 2023 The maln fin8nd81 risk, Bs BW. 18 Iffvnrf1ng Is r￿1 seoJr¢d. In prpvknus ￿#r$ M4RD h8s (eued predoMlr[an￿ on &rtiAI donatiuns and s￿nsOrshIp. Howsv8r d￿rIng 2Q22 tho ¢hadty started dI￿rSity1rg fijnding awnues in Linltr t) reduce risk trorn sptsnsoTS by IdenifyN9 grantmakers fh&l VARD 0)￿d epply lo for th&rtlable nrfing. £6k was seDJred in chariiablg dorratNJns dunno 2022 f(Alovring suThssful applicalsons 8nd further OPpDTkn￿tj?5 are being explored. The Twsle6$ also hav& procedures to alertlhem earty on lo a finnndal short-fall and to act Bppropriatsly dependln9 Dn the siluation. This ￿¢j￿￿e$ a ReSe￿S Poliry 8nd 8tternative anftual budgets io follow based ￿ edLHI (nwme vs requ831ed incomo. In 2023 M4RD rnol hs fuTvJralslng targeL Plans for lutsrn perlody In 2f124 M4RD oins tts defina its next S year5 of strBtÈuy. It w11 no&d tts scaje in ordef to keep up wlh dèmand for de15very (rf Rare Dlsegse ID1 and proyjslon ol expert 8dvlce. By the end of 2024 M4RD ￿rtu have launched Its next phase ol strategy. O¥gantsatlonBI plan5: Rernjft an OpeF3tions & ¥manc8 Manager (Jo McPheTson Mqll then fcus on PaTtherships and Fundrslstng of her Cllrrent mle) ReLYuII a Trainlng PrDgTamTnes Mat￿0er. Rombw the mak? up tsl Ihè Trustees Bosrd and recwrtTrustees based on skill Futuré fundlng: At tho end 012023 M4RD Sent oul fi￿l￿n9 appllcadon3 lo CDrnpanley knvolved kn tho TBre d15ea5e field, eyl8]nlng Ihè thdrit/s 2024 vmrk programma Ind￿￿jnU thè eslimgled Eosts b]vDlved. In lotal M4RD plans kn rd158 £372K ID 8thlève11s alms and obledivesfor 2024. £50K has been cothed overfrnm 2023 and Ihè remalThler bé ra15ed by reathffia QLrt to estsbushed 60urr$, idenltffed grdnl GNerg and 0>pfor￿lI phllanlhropic opporturutias.. Ewents after the end ol tha T¢POrttng porfod Partl¢ulars of eveni£ aft¥ the rÈparbnu date 2re detailed In note 2D lo the finandal slatemen Small Gompany pn)vl8lons This report has been prep8red In a¢o)rdonce tho prowsions appliEdblè to LyJmpanie5 enliled lo th& small DJffp2nlEs eyernption. IS11 1214 Tha trusie8$' annLFd report4WdS èppfDved on 2nd 51gned behalf of the buiatd of tru51ees hy: Mrs L Blnell Twstee and Ch#lr

MEDICS 4 RARE DISEASES STATEMENT OF TRUSTEES, ANNUAL RESPONSIBIUTIES FOR fHE YEAR ENDED 31 DECEMBER 2023 Thg TNstees 8re responsible for prcpartng Ihg Trustees, AnnvAI Report anrl lh¥ financtal slatements In 6cu)rdan( 8ppfic8ble 18w and United Kmgdom Po7JJNting Slandards (Unrfed lfjnudom GÈneraUy Ac£epled knunting Praciice). The law appficable lo thartlles In England and W8les requwe5 th8 TwstÉe3 to prepa￿ financsal 5tal?menls for eath finanripj year vthtth gwe a Irue Rnd fair ￿le￿ of tho stslu ol £ffatrs of tho Ch£rÈty And of the inwnung resourrs and 8ppNraUon of ￿SOm5 oltho Chorfty for thsi year. In prppadno thtse Jlr8nd81 $14lemenls. the TNstees are reqUI￿d to: . sel8Gt sultablg aruuntlng polldes and then App￿ them conslstenyy, - observ& iha rne1hods 4nd pr5ndple¥ In the Charftles SORP; - mBk8ludgernents and eslmates that are reasonab5e and prudent - state Thhether app￿￿bI9 acwunlirg siandawds have been lollo+htd. sublect 10 8ny materf81 deparlures dlsdosed and explained tn the finanaal 51ateThnts' and - proparn Ihe fm8rtC481 ststements on tho gouig ccncem basis unless it Is inappmprtate to P￿suM thBt the c*arty will cxJThthue In Ope￿IDn. The Tnjstees are respons(bla for keeplng sufficlent acajurting ￿￿rdS thal olsr4oso YAtrt reasonable acujracy 81 thna the fvwÉdBI POSLlion of the Chartty and enBble thom to ensure ih81 iho firt&nu&l statements comply wih th8 Chatld83 Ad 2011. th8 Charlty and Repbrts) Regul3Yons 2D￿ and the provl&ons of the Injsl d89d. They Bf8 #Iso responsible Tor saleguBrding the assets of tha Cherity henrs for taking re8songblo sleps for IhB p￿entiOn Ènd deledlon olfrnud and othw bTEgularEties. The INstees' annuat rEPortwa5aFwuved on ...-..-...................- 8nd slgned on behaN otthè beard of trustees by: rs LBi￿rI1 steo IS, 01. 2-Lfv- 10-

MEDICS 4 RARE DISEASES INDEPENDENT EXAMINER'S REPORT TO THE TRUSTEES OF MEDICS 4 RARE DISEASES I report lo Ihe Trusteos on rffy pxamlnaknn 91 thg finanda st8lemcnts of Medlcs 4 Rarq Olse8ses (th& CharSty> for th¢ year endèd 31 Deenther 2023. Respon51blllUes and b4sls 01 report As the TnJstBes ol the Charity you are rosponsible for the preparBlion of tha finan(ial staltments in occordance the ￿￿TeMentS of the Charib2sAd 2D11 Ilhe 2011 Adl. I report Én ￿SPea of my 6XAmin3tiM 01 th8 ChBriWs finanoal 6ts1emenls oui under sethon 145 of th& 2011 Ad. In carryEng OLrt my exBrntnBiion I hav8 foll0v￿￿ ￿1 the appllcable Dlredlon¥ g5¥en by the Ch8rtty Cornm15￿0n undor sedion 14515llbl ollhe 2011 A Independent 8xamInoPa ststoment Your 8ltentr)n Is dravm Its the facl thal the tharity ha# prupared lknandal sl8lements En attordanc8 vdth knuntln J anLI Reportiny by ChBiili&s prèp2ring thèlr accounts In a(￿r￿anCe vath tha Finanaal Reporting Stsndard applicabl In the UK and Repubfic ol Ireland (FRS 1021 In preferenc lo the Poxltthtng 8nd Repoiting by Charfues: Slatement Df RecommeTrded Practic4 issue(l on l April 2005 Is refetrnd la in the 8xJ8nl regulations but has now been %MthdraMTr. l understand that thls has been done kn orderfor finanaal stale￿r￿S ttr PTDvld8 a true and falrwew In ac(ordance swth G&nèWAc¥xpfedAcoJunting Pracuce effethve for reporting peFiods beoinrklng on or after 1 January 2015. I have compIeled rny ex8mtnaUon. I confimi that nD matters have come to my attenuon ￿ conneGbon vlth the èX8minafKll Tne cause to bellesp In arry Mate[1￿ rnspecL' ar£¢LmtsTru rEcords ￿re nol kept In resp6d of the Charityas req￿red by sedion 130 oftho 2011 kl. or nnanctal Btslewents do not accord wlth thoso ￿￿rds. or Ihe f￿and81 statements do not rnmpfy with th& 8pplic8ble fequiffments C¢n￿mIng the fom and o)nlenl of accounts SEI out in th? Charibes (Accounts and Reportsl Regulations 2008 otherthan any Tequlfement that tho 8caunts gi4e a tTr￿ and fawvlth¥%thlrh13 riJta maller ￿n￿dered as part uf an I[￿ePende[￿ exattthiatson. I have no CO￿ernS Bnd hava cyjme Bctoss no other m3itsrs tn connedon the examtnauon ID ¥thlokn allenlion should be tn thi5 report tn otder ID enablv a proper Lmderstandino ¢f the ffnandal sta1ementsto te reathed. Sernkn ShahATtI. FCA SKS Ramon Lee 93 Tabemade Street London EC2A4BA 1510712024 11

MEDICS 4 RARE DISEASES STATEMEIIT OF FINANCIALACTIVIMES INCLUDING INCOME AND EXPENOITURE ACCOUNT FOR THE YEAR ENDED 310ECEMBER 2023 UArèGtrfclod Restrlctsd fvnd? fdnd$ 2023 2023 Totsl Unrastrfctvd ReGtrlctDd fiJDds fvnd3 2022 2022 Total 2023 3022 Not8S ncomo from: Qon8Uons end leg8des Sponsorshlps Investsnents 79.867 164,000 770 79.86T 179.000 TTO 30.083 105,000 100 2.500 10,000 32.583 115,000 100 15,000 Total Inc¢m• 244.637 15,0 259,637 135,183 12,5C#) 147,683 dÉt ChartYAble a(*%￿[)eS 223.107 5,745 228,652 132,627 5,425 136,052 Net Incomg forthg yearf Net movement In fvnds 21,53U 9,255 30.785 2,556 7.07S 9,631 Fund b81Bnces a11 Janu8ry 2023 &5,134 7,075 62.209 52.578 52578 Fund btlance5 tst 31 Dg¢ember 2023 76.661 16,330 92.994 55.134 7.075 62.209 The staierneni of Ilnandal ￿￿lieS Indudes au gBlns and losses r8wnised in the year. Th8 St8temtsnt ol fjnartdal ILli￿tiO$ widudes all oains and lo$5e5 te¢))gnised In Iha year. Ail Inrxme 8xpènditu ¢ertve fmm cbntinutna ga1￿11t$. Ihe ststernenl oi fn8n(i21 dso DJmpfiÈs vath the requiremants for on ints)me and expenTthtsJre actount under the Ch8ridesAa 2011. -12-

MEDICS 4 RARE DISEASES BALANCESHEET AS AT31 DECEMBER 2923 2023 2022 Notss Flxed assets Inlan￿￿18 8ssets Tanglble assels 13 14 2.290 2294 2290 2.658 Current 4SSgts Debtors Cash et b8nk and h hAftd 16 18,925 79.269 5.405 57.787 98.194 63.192 Crtdlic*r5: amounts falllng due wlthln One yoar 16 19,784) (5.9311 Net vmnl aswts 88.410 57,261 Total a360ts 18sy currnnt ITabllltlos 92,994 62,209 Incomè funds Restrfd8d funds Unrestrlcie¢l funds 17 16.330 76,664 7.075 55.134 92.994 62.209 The ts)rnp£ry1s eratitted to Ihe exemptlon from the au(rrt requtremenl contalned In Eectton 14212) ol Charluès Ad 2011, tor Lhe year ended 31 QeLYmber 2023. The 8oxurts hav& been examined by an tndependent OX8minei and thelr ￿POn been tnduded In tnese t7Trandal slalement8. The directors a¢kno￿edg0 thelr respoftsibilittos fttr ttrnptyinu With Ihe requirements of the Ch8Alius ALI 2011 vlth spert tri 8cAiuntoio le￿￿$Slld the Prepara￿on ofllnand81 ststernents. The IlnandBI slatements ￿tre approved by the Trustee3 ori... MIB L Bltrpm Tru$tg9 and Chalr 13-

MEDICS 4 RARE DISEASES NOTES TO THE FINA14CIAL STATEMENTS FOR THE YEAR ENDED 31 DECEMBER 2023 Accounllng pollclo Charlty Infomiatton VÈdlrs 4 R8Tè Dlseases 1$ a pubfi¢ benefjl enlity and a prlvate comparry ￿m￿ted by guarantee. registered tn Enuland artd Wales snd a rruistered in England and Wales. The add￿sS ofthe registered offi¢8 is Unit I2T￿adaWayTethn1taI Centre. Treadaway bmi, Lcudwater, Hlgh V¥onkne, HPIO 9RS. 1.1 Accoununu conventlo Tha t]n&nd81 slBiernents h8ve been prÈportd on tha h15tthlC￿ CDSI bads, es modmed by the rnvaluath)n of certaln finano818ssets and fiabllities and Tnvestsmenl pruperues measwed 81 f8lr vatue thrgugh Income or expen¢JitUTe. Tha ftn8ndal s￿lementS arg prepared In sterfng. Is the fitndlonal uJtTenGy Df Iho entity. Ststsment tsf complIance Trse fin8r¢ti21 have becn Prepared in ￿mpl¢anCe Yath FRS 102. Tre Fin8ndal Reportng stand8rd app11￿ble In Iho UK and the Repubfic of Irelanrf, the Staterneni (rt Recxmmended Prads¢Q 8pplr£bl8 10 (Brhie5 preparing Iheir 8f£J)unts In ¥C￿l￿anC* wth the Fmano81 Reporrirtg Standard 3pplleeblè tn the UK ond Republtc of Ireland {FRS 102) {CltartUes SORP ￿As 102)14nd the Companles Acl 1.2 Golng roncom Thetè 8re no m81eftd uncertth¥es Ihe thorftys to t¢ntiftue. 1.3 Charltable funds Unrestsictsd fvnds are atrailabl8 for USB at iho disuÈbon of th8 Trnslees In furthei8nc8 ol thelr tharitsblg Deslgnatrd fvnd5 8T& urreslrfia8d fimdg eamthed by th8 trus￿eS for pth￿tar prolecl Qr RestrEcted fvfids Bre suble¢Xed t¢ fe#d#ions ort the[re￿and1bjTtr ded6red by thé dont>r or Ihrouuh thh lerffts of An AppeBI. 2nd fall Into f)ng ol s￿￿d￿SSeS. restrtthd Incomg fLmds orendo￿TReIb1Th￿nds. 14-

MEDICS 4 RARE DISEASES NOTES TO THE FINANCIAL STATEMENTS (CONTINUED) FOR THE YEAR ENDED 31 DECEMBER 2023 Atcountlng poll¢l¢B Cont]nuadJ 1.4 In¢omo Ji In(x)tnlng wsuurtss are induded In the ststem8nt olfujandal aC￿￿ties vthen enliuement has passed to thg charity" il Is prob8bla that thè economic benefils 8&S¢ryaled wlh ttke Itensgcjion y￿ll ffow lo Ihe c8rity and the Bmount com b6 rpliabty mSLwed. Tho follovriftg spe(ik appfied lo partlU￿Br e8tpgories of incon￿. in￿me frorn donats"on$ or yf8nts l¥ rpcognised when Ih&r¢ is eyiderte of entittement lu the gi rpcelpl Is probable errfl Its a￿unt c4A be m&8sured TBUabty. leg￿ Inwme Is recoqnlsDd Ythen recelpl18 probablg entitlement15 EstsNlshed In¢J)me t￿rn doneted goods Is n3SWod 81 the folr vaEug of thè goc4s unles9 this Is Umprncilcal to me8sur• tett8bFy. in vknlth c8$0 Ihe ¥8lua is datived ftvm the cost lo Ihg donoror eslim&led rBsa]o vaiue. Donated fÉdUttes and seTrtce3 are rethgnlsed In lh8 accounLq then recefved If Ihe value (an bg reli8bty measured. amounts are induded for tho contlbulion of oenernl yolunteers. . inem frnm contracts for the serki￿$ 15 reo)gnised wtfh the delt¥rry ol the contraded s￿￿e4. Thls is dasslfled as unrethded funds untess there Is a ￿jntrdc1Ue] requiretnenl Ioi it lo bp spenl on g pArti￿lar purpose and rthed if unS￿Al, Sn CAse it rnay ￿ fÈQ8ft1ed as Rstri¢Éd. 1.5 Expendl￿rg Exp6n¢fittEte Is roJgnised on an actruals basis Bs 8 fiabl￿ty Is En￿rred. Eynrfrture InElud&5 any VAT vthlth cannot be futy rno)vered. and Is dwirted hekdings 01 the stsrement of finandal a(thltres lo %thlth It relBles-. eypenrfityre on raigng lunds indud6s the ojsts Df all fvndBising acti￿)es. events, norK8tttable Iradlftg 8dvJttes. and the $818 of donated gcods. • eK>en¢fitrJJB Dn ￿￿1￿blO 8cilvitles Indud•s ail costs ￿ttJITed by a charity In undertaklng athllles that furtthtr its charitable Blrns for the bÈn6fi1 of its benefic33rfgs, Indu&inu those support ¢£+sls end Costs retaung to lh¢ govemanco of tho chadty Epportloned lo tharitable adlY1t￿s. ther expendltrrd Irdudes 4U eypen(tlure Ihal Is nellher related to rals5ng fund5 lor tho chartty nor part of tts éypenditsr8 on charilabla oththyes. Al cos15 arB ollocaled tD e4)en￿l￿re Galogjrfos ￿￿O0￿g the usa of tha tesourr£. Direci c•sts attribtslablè ta a gnole athwty ére alloGa￿d dltedly to that a￿vIly. Shared cosls are apportioned behyeen the adI￿￿eS they ntnlula tn On a rea>)nabtB. jusltffabla And cxjnsisteni ba&s. 1￿ InlaTrglble Il¥&d oxsèts othgr than goortwill lrttangNe a￿1 aquired on accounl ol reglstrauon of trodemth & logo ts reco3onlsed at ￿st Inpa￿ad on fj]Ing thè patenl YAlh the competent authortiy. There Is no Amorusxlion tslaffg8d for inianubi le aojuifed on 8C•X4Jnt DI registralton of Itadem8ik & Logo. The carrytng value, hMv8r I￿1 b8 tovtewed al Bath Bafance sheet dala.11 an enuty detettnno5 that Ils tradernaths Is wjrlh less thafj it wa$ 8 yaar allo. th8 of th6 Intangibk assÈl must bo impar¢(t. WThan an impaIm)énl ooxrs. the Val￿ of tho assel must be deLYe8sod to Ils current m2Fkèt fdluo. Th8 diff8rencè befyswn the ￿rrenl Valuè of thè trademark its fomiervatue mu5tb8 recorded as a Ios5. 15-

MEDIGS 4 RARE DISEASES NOTES TO THE FINANCIAL STATEMENTS (CONTINUED) FOR THE YEAR ENDED 31 DECEMBER 2023 A￿OUn￿n￿ pollclB5 (Contlnuodl 1.7 Tanolb18 ftxed 8ss0ts Tangli Jlc assets ar8 Irulafy le￿rded at cosi sub5equentty 515ted at ￿5t less any 8c£umuIatttd eprguabon and iry2im)enl losses. Nny ￿ng￿l le assets ¢SJtt¢d al t¢vduf3d 4unounts are rECo[d￿ al kn fair vglug 8t date of rev8luolAon less any subsequent acurntslaled dopr¢dation gnd subsequ£nl 8lKJJffi￿1pd tThpal(rnenllos5es. tnu885a In th& eAny7nu amount ot An 8ssèI as 8 result ol.a rov8lua¥on. Is reo)8nlsed In othBr recognised uelns and losse8. unless ft roverses a thBrg8 for tmp81rment that has pre￿(￿SlY b8¢n ￿¢0snISed BS e>pendiknrn ￿thIrt the ststefflenl of Ilnand81 adi￿U03. A decBase In Iho ra[ryI￿g omDiJril of on asset as a re5uIt of rer4lu8tion. Is recognlsed In olher recogn5sed gnins and losses, except lo 1shli 11 offsets p￿0￿8 (Bvalu8tJun y8Tn, In c8se Ihe 105s 13 shown Ydlhin olher recognlsed 081n$ and IossBg on the statement of finandal 8d1￿￿e$. Depredatlon is ￿(x)gnISed so as io wfte off the or Yalualon of assets less theirrosfdual values 0￿r their usefvl Eves on the fdlowinll bases.. Computers 2$% Redu¢xtg B￿ance method The g81n or IOSS 8¥t51ng on Ihts dl¥osal uf 8n 8S5el Is delem)tned as the drfleren¢¢ be￿￿en the sale pr￿￿ed$ and the Cl￿ng yalu8 ollhe and ig ￿e0c￿E&ed in the slatomenl 0ffinand81aL￿ts'65. 1J Impalmiont offlxèd Bssgts Arc￿eW for EndiG&lors of iThq)airmentl# raifed eath reporting date. ¥￿1￿ the rnfxver8tle afftoumt betr esumated vknere suth Indlc4iors gyJsL IAhete thv orrylng vg]ue exceeds tha rec4ver2bla amount, the assel Is impairEd 8¢¢0rrfJnBly. Prior irrp8irnents arè also reme￿￿￿ fDrpOs&￿Ie Djyersai at eath reporbrbg date. For the pwpvses of tmpairrnent leslirTg. li 15 not PQ55ibte to estimate the (ècovtr8bl8 WVOUnt ol Bn Indi￿dual asse{ an esttmate Is made ol'the reojverable 8mouniof Iho cash-generatlng unlt to thlch the asset belongs The caenerabng uftii Is the sM￿leS1 idenfjft8bl8 QTUUP ul assBts that indude5 the asset and generntes cash I￿Ow3 th81 lerqety Independenl of the cash tsthows from other a$3ets or gr0￿p8 ol ass8ts. 1.9 Cash and cash equlvalents C8sh grhd tAsh equtvalents Incjude c2sb In h8nd, dÈpolts held at caIl ¥￿th benks, other Short-le￿ lI￿d Inv8slments with Ortgin￿ msturilTeG ol threa rnonths or lesg. and bank overdrats. Bank ove￿18ft$ arts 5hD¥wn ithln t0FT0￿lngS In c4rTheni fHbl]itles. 1.10 Financlal Instruments Afin2nd81 assei or a ffnancjal llabitsty Is ye￿an[Sed orjtyvthen the thaTity becomes A party to the c>Jntraraual provlsions o1th8 instnment. Basic fin8ndal InsinEments art tnlltaty tr>Jwlsed ot tho amount re￿Nable or payable indu¢fina arry rÈla￿d tJansaclion c4sts. Currenf 8&setS 8rtd ￿Tran1 llabiKties are subsequenty M￿sUre￿ at tha cash Dr other ¢>JnslderaUon tsxpecled lo bo pald or rEC￿￿d and nOtth$4￿nt￿d. Qobt Ins*vnants aro sub￿quen￿Y rn88SUTed al 8mortlsed u)sL Svhere Inveslments Tn th8res erv pubEdy traded or th8￿ falr valu8 e2n Mèasyred ye￿ablY. Ihe In¥e51menl 15 sub5equenlJy rne85u￿ at fair vatue trlth thange5 In falr value recognised lft In(>Jma and expen[￿[9.AI ¢)ther Investmsnts wbsequenty measutEd otujsiless ÉmpalrmenL 18-

MEDICS 4 RARE DISEASES NOTES TO THE FINANCIAL STATEMENTS (CONTINUED) FOR THE YEAR ENDED 31 DECEMBER 2023 Accountlno pollclos {Conttnu8d) Other finandal Instruments, Induthng derlvaovos, are In1￿￿ty reo)gnlsed ot falr wdluo, UTpless pa>Thnl fDr awl is defrtTEd beyond llL¥mal business fem￿ or financed al a ral& ol itiletsst11g1 i8 not a matktl rèlé. lo whlch cas& lh¢ asset is measured at the present vahje ol the ftkn payTnenls (fiscnunled Bt B martet rale of interest for8 SI￿alar debt Ubs1￿Jrnenl Other finond&l llknents arn gJbsequ2nty meBsured al f&r Va￿e. Kith llny chenges reo)gn15ecl In th8 statement of finandal 8thUes, vAth the exception of hedging Instruments In a deslonated hedgfng lationshlp. FEnÈnci81 a£￿ts th2t ar8 nJsurèd al cfjst or 8morfised o)5t em r81iewèd for oblpctive ov1denc￿ of rmPÉl￿anf al the end of esth rnportkno dalo. If ther6 ts oblecffve evldence of 1rnpalm￿ftl, an Impar￿Ne￿I l95S ts Jecognised under fha 8pproprlate heAOing lrt th8 stslementof fu)Bmclal acUMYes In ￿lch tho tniUBI gain was For all equty kjstrumenb reoardfess of slonifranc4, and other f￿an¢)8[ assets that ate in¢thuduatty stgnMcanL eso arè 2ssessed irtdividuaLW for impairThnL Other fjnanda] 8ssÈts arv either assessed tndiwdLtalty or gtDuped on Ihe basls of slmlar c¥edit risk thataderfslks. Jw reversals of brq)airment are TrayJnlsed Imrnediatety, to the ejlent that the reveTsal does not resuli In a nying omount of th& fingnaal 8ssel thAt wAceeds Ba￿£ flnanclo18SStts Bask Ifnandal assets. Sndude debtors and cash And bank balances. are Inltl81ty measured 21 irans8caic+n price Fnduding trans8cUon c4s1s end ar6 subsequenty cerri¢d É18rnrt1sed cost usb)g the effeclivo interesl method unless the arrangement ￿nSIrtu1e5 e tr8n3acllon, the ￿nSe¢ll0n Is rrftBsured al Iha ptesenl value of tt)o future receipts ￿LS￿￿nIed a18 market rnie of Interest. Finaftdal 8ssets da5sffied a5 ￿￿1Vable withtn one year8ro not atnortised. Baslc ffnanclalliabllllles 88slcfin8nd¥l L4bPilies, credllors and bank IDarn are knttlally rec¥)gntsed 81 iransacbon prfc unless tho BrTangement constilLtles 8 thAnting transathon. ￿ere the debt lnStrL￿e￿I is measwed at th? presenl valuvj of Ihe pa>Thernts dkuyunted at a matkel rdle ol InleresL Fln3ndd ￿abIlitieS da53med a5 payable vAthin one yeArare ntsl aTfv)itis¥d. Debt in5truTTrsnis Bre subsequartty carrled al arnrtised ts7sL usng the eifedtvg interest Tate melhod. Tr8tl& a￿ oblig2livns to Pay for gowls or 50r¥Kos thal hav8 been 4(4uifEd in the rKdinary eLyJrsè of operauons fvam suppfjers. ArnounLs payable are da5sffied ay gJrKent llablDtlcs K Pa)￿nI Is due fAlhln orÈe Yar Dr 12S5. If not, they ara presented & rton<yJr￿nI tabifitivs. Twad? r3BdiiDts are reccgn5sed ￿[traI]y Bt an5aclEon prtceand sub5equenlty measured al amortlsed C￿$1 uslna In8 effeth Inteft Method. D¢tEcognldgn of Ilnanclalllabllltles Ftn8ndal Ilablllues aTe derectr3n15ed Ihe ChaiiW8 ￿ntra(￿jE1 obllgailons eyplre or arn rftsolarued or canc*lled. 1.11 Employ0& bonsfjts Thg Cost ¢1 any unused hofd4y enuttemenl Is rgcoanlsed In the perfod In Iha employfjo's servlcos ar i¥ed. Tenrunation benefits arè reco￿18ed immediaiew Bs an 8xpens8 vthen the Charfty is den￿nSt￿dbrY commilled lu tenminala tho oMpl￿￿ment 018n emplo￿ or ta prDMdg knTllnafk+n tenefits. 1.12 R&Urement bonofils PwThnts to definèd ￿r￿￿+vu0ft rets￿Ment tenefft sthmos ore tharoed as aft eJyens8 89 thtyfall du&.

MEDICS 4 RARE DISEASES NOTES YO THE FINANCIAL STATEMENTS (CONTtNUED) FOR THE YEAR ENDED 31 DECEMBER 2023 Aecountlng polk18S Iconttnuedl 1.13 Fund accGunttng UnresftKa¢d frJnds a￿ Èmllabjc for use al thg dlsitetion of the tJ7JStees ta {￿ther 8ny of the that1￿5 purposes. De31onaled fvnds Orn unreslrided funds e8Jm4tktsd by tho truslEes for partIc￿l￿r futuro proied or crJThthmerrt. Restsidad luftds a5v subJecled ID rostrtcllon3 on thelr e￿endthrB dedBrtd by lh8 donor ¢r tjwotsgh thFJ lewsns ofan 8ppeel. 8nd fall inlo ong ol Sul￿d¥s$05. lestr1￿ed Inc4me funds or endo￿OnI funds. 1.14 Deflnad contrfbuuon plans Contribuuons to d•fjnad ¢ontribution plans Èrt recugni5ed us 8n expens¢ in the perfod in Mthlch IhB related setrfc• Is p￿ded. Prepsld ctntrftrxrttons are rÈC￿nISed as an 835et lo the exteol that the pr8payThnl lead to s reduction In ￿t￿r0 P8yTrents ora cash refund. en o)nlributions 8re not &xpe(aed la be settled thcljy within 12 motths of the end of the rèportino date In ch the ern￿OyeeS render the related Sth￿. the Uabllty Is measured r)n a dtSCOLmied presenl value b2SiS. The unvlnding of tho discL)unl 1$ rncogftised Ès an e>pense in the perlod tri it arise& 1.1S Llmlled by guardntso Medics 4 R8re Olsease Ltd Is M rajmpany ItrT¥led byguaranlee and according￿ does nol have 6 sh8rv cepltd. Ev8ry m8rn￿r of tho company undertakes lo c4Jntrkn suth amouni ks mty bé required not exwding £1 to the a55els of the thwilablg company tn the evenl af Its belng w)uDd up rfle he or fhe Is o rnember, OT thtn one year 8fterho or she ceases lo be a mernber. rltl¢al tsG¢ounUng esumatss and ludllernents In Ihe appDcabon of Charitys 8cuNnling potides. the Twstèes srè reqtsitsd to m4ke judgements, estlmates and 8syJryYon5 8b)Ul Ihe rarrylng amount of assetg and labfGttes that Bre not rnadlly app&￿nI fmm other souffes. The estimtilès Bnd assouaied 4$suM￿On5 arv b8sed on historic￿ 8XPe￿8nct ond oth¢r fadors fhal ￿nSIdered to bo releyanL re￿￿15 m8y dtt[erfT￿ll these eslimaies. The esiimal85 and undertythg 8ssumptfons 4re re￿0￿￿ on an onuohu basts. Re¥5slDn3 to acuunting estim81es Ar8 fECXSGThsed in th8 wiod ift thB 8tmat8 Is twlsed the re￿￿On affetts only th91 perfod, or In thg perfod of the ￿￿$3¢n and ftthre pertrxls ¥￿ere th8 Te￿Slon affects both current ond ftjture pellcrfS5. CrftlcalJudgements Tho PTeparnllon of the ffnancaal slatements requires Fn8nagemenl to make ludgernents. estrnaies and as5urybon5 thal affelt the amDunts reported. These estimates ludgements ero ￿n￿nU&,1Y r¢iri?wed and ba￿ on •xp¢rt8no and othei factorn. Induding o>wAatlans ol Iuiure evenls th61 arb bO￿Ved to be rÈasondble under fh8 d￿M&18n￿> -18.

MEDICS 4 RARE DISEASES NOTES TO THE FINANCIAL STATEMENTS (CONTINUED) FOR THE YEAR ENDED 31 DECEMBER 2023 Danatlons and léua¢los UnTe8trlctod Restsictsd funds nds Totsl Unrestrf¢t8d R8stridgd lunds funds Total 2023 2023 2D23 2022 2022 2022 Dorkatsons oifts OthBr 46,287 33,580 46287 33,58Q 20,S56 9,427 2,500 23.1S6 9.427 79.887 7g,867 30,083 32W3 In￿me from charltablo actlvlUo9 Unrestrfcted RestrS¢tsd full￿ 2n23 Total UnT8Strf¢led Rostrltted funds runds 2022 2022 Total 2023 2023 2022 SpDnsorsMps 164,000 16.Q()J 179.OQO 105,000 10.000 115,CpXI In¥èsbngnts Unrestsletsd UnreJtrf£tsd funds funds 2023 2022 Inle￿$1 recelvable 770 100 18-

MEDICS 4 RARE DISEASES NOTES TO THE FINANCIAL STATEMENTS {CONTINUED) FOR THE YEAR ENDED 31 DECEMBER 2023 Chatifable acd¥lUes Actfvftt85 undert4k¢ n diTretty Support oosts Yotal Activltlos 2023 undgrtake n dlrec Support costs Totsl 2022 2023 2023 2022 2022 Stsff ojsts Depr¢d8tlon Renl Insurance Telephofte Compuier vxpensos Subsurptions Interest pald B8nk¢hafgas Admlnlstratlon support Advertlslng 8t￿ rnartejjng Evenl costs Trdvelling and sUb￿tenCe Postsgo 8nd Print Consu113nCy Adwlnlstralive Supporta VleW&re ijther Cosi 156,077 732 6,SSO 438 48 7,615 169 156,077 732 6,590 438 48 7,615 159 103.233 487 1.460 247 12 3.426 170 103233 487 1.460 247 12 3,426 170 66 275 11,879 8,435 11,502 1.346 14,783 56 275 11,879 8.435 11.502 1.346 14,783 142 322 18.752 66 2.436 675 $40 142 322 18,752 66 2.436 675 1.744 113 1.744 7.163 2.696 49 2.6g6 3.385 7,050 a,336 221.802 7,050 228.852 134.716 3.a36 138.052 Analys15 by fund Utvestrf(*ed fvjnds Restslcted funds 216.057 5.745 7.050 223.107 5,745 129291 5,425 3.3￿ 131627 5.425 221,Bu2 7,050 228,852 134.716 3,338 138.052 Support ¢osts 2023 2022 Independontexamlnatlon Other 85gJranco seThTCgY 1.320 s,rJo 1,200 2.136 3.336 -20-

MEDICS 4 RARE DISEASES NOTES TO THE FINANCIAL STATEMENTS (CONTINUED) FOR THE YEAR ENDED 31 DECEMBER 2023 Ipt rnovomant In fund8 2023 2022 N?I m0￿ment In fvndj is staled Aftèr thRrgittllllrJediUng1 Dep￿09￿on oloymed tsngiblè ftxed 85£4ts 732 Yrustsès NDng of the Tru51eB5 {or ary Pgtsohs Ll)nneGled ￿th them) recelved ary rrmuDerntion or benBfits from the ChÈrili tlwfng the year. £90012022: £3001 Wds pafd to Mr D Jeffrfes. one ol tho Injslees, lor hls professlonal seThi￿$ In Éètbng up e leamlng fadfiffes for thg tharf 10 Empfoyegs The ev8rag• nTh3nVty number ofempknyees dwirwJ tha ye¥was: 2023 Numbor 2022 Numb8T Chariiable ÈeJMUes Emplaym•nl costs 2023 2022 Wages and saTarfes SoeAg1 seuJJYty tfjsts Other penyon ￿$1$ 144.715 8,277 1085 98.105 3.085 2,043 156.077 103,233 Out ofth? abo￿ enwioymenl ￿$1, (%15tamuunting tg £4,845 (2022: 4.468) lom￿ p8rtof restrfded 0xpendi￿re. Thernknttt no employees vfvse annual Temur￿ralton Wds rnorv than £60,OJO. 11 Pen31on¥ and vthor post mtlTvrn¢nt benofitS ts8llnod Mntrlbutlon plan8 Tho amounl ro￿￿n[sed in Incoma oraxpenrfElure as all 8xpense In relatlon to defmed ￿nI￿lA￿lOn plans vrds £3.086(20￿ £2.LW3). 12 Taxall4)n Thp 15 exempt frcmtaxaton on its athNi¢s BII ts ino)mg1s ¥pliedforalat5tabJa Pu￿0￿e& -21

MEDICS 4 RARE DISEASES NOTES TO THE FINANCIAL STATEMENTS (CONMNUED) FOR THE YEAR ENDED 31 DECEMBER 2023 13 Intsnglblè fixèd 48s&ts inlhnglbl•sAu4ts Cost Al l January 2023 and 31 De¢¢ml)er 2023 2290 Amortlsatlon ehary Al1 January 2023 and 31 December 2023 Carrylng arnount At31 QecemOer2Q2J 2.290 At310ecernbcr 2022 2290 14 T4nglbl8 assots Comput•r• Cost l January 2023 AddlJons 3,964 3B8 At 31 D￿rnber2023 4,332 DeprecTatloA Al 1 j￿r￿ry 2023 Depreciation (*arlled in lh# ye8S 1.3 7Y2 At 31 December2023 2.038 C8rrylng amount A131 Derxmber 2023 2.294 A131 Detsmbor 2022 2.658 16 Debtars 2023 Amounts falllng dué withlrt one ￿ar. Trad8 debtor8 Ptepayments end accrned 13,643 5282 300 5.105 18.925 5.405

MEDICS 4 RARE DISEASES NOTES TO THE FINANCIAL STATEMENTS {CONTINUED) FOR THE YEAR ENDED 31 DECEMBER 2023 18 Clodltors: amounts lalllng duo wlthln ong ￿fjr 2023 2022 Other taxabn and SOL?al sewiity Trede >ethloTS rrJJ815 And dBfeNed income 3.413 3.429 2.942 3.077 2.620 9,784 5.931 17 RoBtrIcied fund ststcled fitnds ol thg tharity comprise the Une￿ended bal8nt8s of and gTrnts held on Iruzl subfv¢a lo spec4fic r￿nd[￿On5 by donors as to how they msy b8 use0. At 1 January IncDmEng Resou￿e8 2Q2J r¥sDurces Qxpended At31 December 2Q23 M4RD LÈam 7,075 15.000 (5.745) 16,330 Piavloug yAar. Al 1 January 2022 In¢omlng Resour¢¢s èxpend At31 Docember 2022 M4RD Leam 12,500 (5,425) 7,075 18 Unrestrfelod fund• The unrÈstsfthrt ftjnds of the comprls6 Ihe uneyended balancey of danattons and Erdnts are not sublect ts spedfic conthtion5 by donors and 9rantors as to howthey may be used. Th6se Indude des￿anated fvnd5 have been set a8lde out Of untestrkted fvnds by the trusteE& for speejffc pU￿oses. At1 January 1023 Incomlng r8souw R•50urcps expendod A131 December Z&23 General fiJnds S5.134 244.637 P23.107) 76,664 Prvvlous yoar. At 1 January 2022 InE¢>mlng resou￿￿￿ Royoufcej JYPgnd At31 Dge8mber 202Z Ganeffal fifftds 525T8 135.183 1132.627) 55.134

MEDICS 4 RARE DISEASES NOTES TO THE FINANCIAL STATEMENTS (CONTINUED) FOR THE YEAR ENDED 31 DECEMBER 2023 19 kn41￿1￿01nOt aSY8ts batwon fvnds UnTOStrf¢ied RDStrfcted fvnds fvads 2023 2023 Total UnroAtrlcted Rèstrlcl8d fvnd$ 2022 Total 2023 2D12 2022 Fund lJglanc£s 8l 31 Dectrntbef 2Q23 represented ty. Intartrgti 18 fued a$5ets Tanoiblg 83sets Curreni 8sSet&iifiab￿ltieSj 22 2.294 72.080 2,2gD 2.294 88.410 2.290 2,658 SO.188 2290 2.658 S7.261 16.330 7.075 76.664 16.130 92,994 55.134 7,075 62,109 20 Relatpd party transactlons Duting the ye8rthe Charity entered tnto the fdlowing trBn4dons ￿71￿ related p8rttèS: Donabw of£Na p022. £4851 receNed from Luw klalha Rosemary Mtho is colrow secr813ry of charity. ConsulSng fees 01£600 {2022-£3001 pald to Dan Jelhlèsvkno Is tuslees of charity.