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REGISTERED COMPANY NUMBER.. 10646129 (England and Wales
REGISTERED CHARITY NUMBER.. 1175311
INTERNATIONAL NIEMANN-PICK DISEASE
REGISTRY
IA COMPANY LIMITED BY GUARANTEEI
Report ofthe Trustses and
Financial Ststements for the Year Ended 31 March 2024

INTERNATIONAL NIEMANN.PICK DISEASE
REGISTRY
Cont¥nts of the Financial Statements
for the Year Ended 31 March 2024
Page
Rèlerènce and Administrativè Detalls
Report of th& Trustees
Independent Examlnels Report
Statement of Financial Activltles
10
Balance Sheet
Notes to the Financial Statements
12 10 18
Dètall•d Statement ol F￿nanCIal A¢tlvltles
19

INTERNATIONAL NIEMANN-PICK DISEASE
REGISTRY
Reference and Administrative D8tai15
for the Y8ar End8d 31 March 2024
Trustees
J E Green
T Hwot
Mts T A Malhieson
Dr A Dsrdis
Dr M Vaniei
CtroptedTwste8- G￿lty1
Company secretary
Mrs T A Malhieson
Reglstered offlce
Suitè 2 Veirnont House
Washington
Tyne and Wear
NE37 2SQ
Reglstered Company number
1C646129 (England and Wales)
Reglstered eharfty number
1175311
Independent examlner
James Anderson & Co
Chartered Accountsnts
Pentland Eslale
Slraiton
Edinburgh
EH20 9QH
Chief oxecutlvB officer
Conan DonrEIIy
Page 1

INTERNATIONAL NIEMANN-PIGK DISÉASE
REGISTRY
Report of th8 Trustees
fortho Year Ended 31 March 2024
The trustees who are also director5 of the Charity for the purposes ol the Companies Ad 2006, present their report with
financial slatements ol charity for the year 8nded 31 March 2D24. The trustees have adopted the provisions ol
Accounting and Reporbng by Charili8s.' Statement of Recommended Practice 8pplic8bb to charities prep8ritvJ their
accounts accordan[￿ with the Financial Rèportirg Stsndaid applicable in tIE UK and RepublTe of Ireland IFRS 1021
leffeclive 1 January 20191.
Objectives and activities
Backgiound
Th8 International Niemann-Pi¢k Disease Registry IINPDRI is an ind8pendant. not-for-profit charitable company
regiSt8￿d in the UK Cleated by a collabofalion of palienls. health professionals and medirAI iesearchers. it offers a
single. di5ease-specific global regislry data r&sou￿ that indudes both Clin￿81 data and patient reported data for Aod
Sphingomy81inase Deficiency IASMDI Niem8nn-Pick disease. and Niemann-Pick disease type C.
Due to its rarity. r8gi51ration for Niemann-PKk dk8e8se5 is partKularfy important. Registration can provid8 Sufficient data
lo understand its cO￿p1eX natural history, support acc￿18[ated recruitment to trials facilitate the devdopment and
monitori￿9 of care standards.
Mission, Vlslon and Princlples
The mission of Ihe INPDR is to document the Ni8m8nn-Pick patpnl experieTh￿'. to improve diagnosis, trealment arKI
cai8, to advance r8search and ultimat81y, to improve health outcomes for 811 those affected
Our vision Is the cr8akn.on aThS ong(Mng devdopmenl of a comprehensive. international (J8ta resource, sperific to
Niernann-Pick diseases which benefits patients by increasir¥J understanding ol these rare conditions, en(x>urag&s
effluent and timely diagnosis. enabtes progress in research and clinical Irials. and laulitates the development of
therapeutic in18tventions.
INPDR advan¢es ourwsion by adhering to following prin￿￿es.
owe respèct and protect patient piivacy, arKI build trust among our patierit c(xHmunity.
o We striv8 for excellence through ¢￿tInUOuS improvement. professionalism and expert support.
o We operats lo the hvahest leve15 of suentific integity arKI promote high-quality research.
o We a￿ transparent in communilxtions and lake respoTrsibilily lor our action5 and decisions.
owe foster an environrnent olcollaboration to itnprove outcomes in Niemann-Pidt diseases.
o We embra¢* Innovation and change as appropriate to our need$.
We shall remain indetend8nt of ￿Voca¢y, policy and commercial interests and bl the data tell the story of
Niemann-Pick diseasès.
A key strength ol the INPDR is the collaborative relationships developed trom the outset and enabling the powerful
combination ol patient ￿adershiP and professional imp4emènialion. This ensures the leort5macy ol the patient
perspective and ensures Ih8t il is at the heart of our organisation and mission.
Page 2

INTERNATIONAL NIEMANN-PICK DISEASE
REGISTRY
Report of the Trustees
for the Year End8d 31 March 2024
The INPDR will work to eThh8n￿ and fa￿lItate progress through the following airns and objèctiV8S'.
Alms
o To build a high qu81ily, complete Inleinalional populalion-based Niemann-Pick Disease Registry
o Proted patient privacy and ènsure (xrnpI1an￿ wth data protection an£J the highest Clinical stsndards
o To f8cili18te clinical rtsearch through data sharing provision ol analytical 8eNces
o Provide evidence to facilitate advocacy and inform publK debate
Objectlves
o To describe Ihe epidemiology and natural hislory ol ASMD Ni8rn8nn-Pid( diseasè and Niemann-Pick Typè C
o To SUPPDrt development of st*ndaids of diagnosis, clini(xl rnanagement and quahty ol care
o To e5t8blish genotype-phenotype correlat'ons
o To piowde support lor equal accB$5 to biochemic81 gr￿ gerElic testing
To faTrlitate accelerated r8(Yuilrnenl to intetvention81 st￿lIeS
o To prowide a regulatory compliant registry 10 satisfy posl-markeling oblvJation5
o To support regulatory interaction between nalionallsupranation81 rnedicines agenc￿$ and ph8rmaceutical compani85
o To a￿lerate progress in underslandiw NPD, by encouraging 8duc2tion ol health professionals and researchèts
Background
The INPDR is a regulatory-compliant. secure dalab8se ol Consenting pa118nts, with appropriqte security, manageffl8nt,
and controls. A secure web-based electronic dats capture IEDCI ￿8￿orM is used for the 5yStemalic and uniform
collection of clinical d8ta ol patients diagnosed with either Acid Sphingomyelinase Defiaency IASMDI or Nlemann-pick
Type C INPCI. The 8clive participalion of clinicians and pabents from around th8 wodd facilitates the collection ol
demogfaphie inf(Kmation. diagnosknc ie8ulls, treatment, 8nd h8O￿h outcomes data The INPDR is a long-temi initiative
that will continue to grow and to create mechanisms to ensure that the data (x>1￿ckd woiks hardest to 5erye patient
needs.
Siqnlll¢ant activitie5
In the 18st year we have worked to implement the INPDR'S global reciuitmenl plan, lo acc818r8te data collectK)n,
improve clala quality assurance and to facililaie research with Ihe ultimate goal ol improving outcomes in Niemann-Pick
diseases,
Clinlcal Data Colleetlon
In 2023124, INPDR collaborated wth 22 clinical site8 across Europe, North Arner￿a. and South Arnerica to collect data
on 450 pstients 1350 with NPC 8nd 1C() with ASMDI for clinical research. Th8 r89islry is expanding with 44 new clinic81
site5 In Europe, Australia, and th8 US.
Patlent Reported Dats
A revised pat*nt-reported database IPRDI launched in 2023 saw 144 patients enrolkgd by March 31, 2024. mathng a
40¥. growth. Vvhile growth has b&t*n slower than expected, Improvements including promotional aclivilies and user
interfats enhan￿MentS We￿ Inilialed. By March 31, 2024. several qUest￿nnaireS were completed, including 96 quality
ol life foFrn5 and 102 symptomslsev8rity assessment5.
The PRD is currently in English, bul a mulb-language solution is in develoKKnenl for 2025. Data quality monitoring was
strengthened in the US and UK. SUPFK)rtaY by the Ara Parseghi£n MedicAI Research Fund, wrth wsits to clinical sile5 to
ensure data accuracy INPDR also achieved IS09001 a¢credilation for Qualty Managem8nt Systems, confirning ils
strong registry and 18588rch support cap8bililK95.
Page 3

INTERNATIONAL NIEMANN.PI¢K DISEASE
REGISTRY
Roport of the Tru8tee5
for the Year Ended 31 Mareh 2024
Obje¢tive8 and aetlvltles
Anonymlged inforrnatlon $harlng
INPDR suppNted multiple research projects focusing on various a5PeCts ol Niemann-Pick Disease, Including disea88
pl0g￿$s1on, qu81ity of life for patients 8nd carer5, seizure burden, and the management of associ8ted conditions like
infiammatory ix)wel di563se.
S8v8ral antsnymised d8ta requests were pro￿Ssed, including genotype reports for the NPC population. 8n anonymized
dataset was prOV￿ed to C-P8th, and a ￿VIeW ol the All-stripès database to assess ils ulilily for a Us-led sibling study
was undertaken.
Publlcations, Reports and Poster8
INPDR were also invofv8d in reports and publicth.ons Induding, a 2024 publication in Orphanet Journal ol Rare
Di4e858s expk)ring the re81-lif8 impacts ol olipudase alfa on ASMO patients and their families. In add￿110￿, INPDR
published both quanlilalive and qualilats.ve reports on th8 impact ol olipudasg £lfa on adutts with ASMD,
contribukn'ons from INPDA, NNPDF and NPUK.
INPDR also contributed to posters al several cOnfe￿n￿$ indudiry update5 on Niemann-Pick Typ8 C and ils research
al the WORLD Confer8ntE in February 2024. including findings on impad of olipudase alfa on 8cSu115 with ASMD
and real-world study from the Early A¢￿$$ Program with Arimodomol.
Public benefit
In shaping our objectives for th8 yèar and ￿annIng our activities, the trustees have con&de￿d the Charity
Commission's guidan￿ on publi¢ be￿fil. including the gUidan￿.pUb1iC benefit.. running a ch2rily IP821'.
Achlevement and performance
Charltsble a¢tivltles
Grant funding in Ihe form of unrestricted educats'onal grants enabled the charty to carry out ts acliwties. In addition,
INPDR Gateway provided payment io INPDR lo facilitate its ser￿￿$. Further funding came trom the Ara P8r58ghian
Medral Research Fund.
Vve have conlinu8d to maintain a strong pr85ence in the field of research have collaborated on a global basis W￿th
other rare disease oiganisalions, research and academic institutions, regulators, and industry to pri￿[@s$ and further
dgvalop the registry, and to faalftaie research and diniral development.
We continue lo sustain appropriate relationships wth the pharma￿Ul1CaI industry, particulady those eng￿ed in elinieal
ogiarnmes and activities in the field of Niemann-Pick disease5 and lo work dosely with the global patient advocacy
leaders supportitvJ the N￿M8nn-p￿k community. This indudes the International Niemann-Pid( Dlsease Allian￿. whos8
presenlalives ale appointed as INPDR Board and Scientific Advisory Comrnittee Mernbers.
Wè continued to engage with the global Niemann-Pick community through ￿irtUal in-person conferen￿$ and focus
groups.
p￿e4

INTERNATIONAL NIEMANN.PICK DISEASE
REGISTRY
Report ofthe Trustees
for th8 Year Ended 31 March 2024
Achlevement and performanc¢
Achlevement and perfomTrance
In the last year we have worked to impkment the INPDR'S global reeruitm8nl plan, to a￿￿rat8 data colledion.
improve data qualty assu[ant￿ and to facilitsle research with the ulllmate goal of improving outcomes in Niemann-Pid
diseases.
During 2023124 INPDR worked with 22 clinical siles in Eurq)e. Norlh America, and South America to collect clinical
data on 450 patients that will be used to support 8 r8ng8 of clinrAI research studies. This included 350 people with NPC
and 100 with ASMD.
With increasing engagement from the global dinical community, we are slso onboarding a further 44 clinical sites in
s8vgral European countries, Australia, arKI the United Slates. This L% the first step toward growing the ￿giStry and
(aplurirvj cntically Important patsent d8ts.
-The registry a150 10118d out a revised patient-reported database IPRDI in 2023. He￿ patients can share thèir
experien￿ of Ni8n18nn-Pick Disease through a seri85 of questionnaires. There were 144 patients in PRO by March
31st 2024 133 people affected by ASMD ancl 111 with NPCI, over 40% growth on th6 pfftwous yèar. lllcieases were
ObSer￿d across sever81 regions but ￿r￿CUlarty in US. Europ8 and UK.
However, gr￿￿h of the PRD has been Slower than anlicipaled. Consequently, a number of improvements were
initiated during the period including a range ol piomolional activities and user int8rf8c8 enhanc￿mentS.
Not all people ￿gistered in Ihe PRD have ¢ornpleted oueslionnsires and som& h8ve CoMp￿ted questionnai￿$ acr055
different lime points.
By March 315t 2024, 96 quality ol life questionna1￿5 have been completed 181 NPC & 15 ASMDI. 78 Caier qu81ity of
life questionnaires h8ve been completed and 78 NPC quality of life forms have been eomplèt8d. 102 syrnptoms I
saveiity qu8Stionnaires have been completed186 for NPC and 16 frir ASMDI.
Vthile the PRD iq currently available in English 18ThJuage, work has progiessed swn1fican￿Y with our te¢hnolcvJy
partner. OpenApp, to develop a mulliple-langu8ge Solution wth the expectation that thi5 will be iolled out in 2025.
During the year. we embedded our d8t8 Quality monitoring function In the US 8nd UK with the support ol the Ara
Parseghian MeLlical Research Fund. Our Clinical Research Assooat8s continue Io wi51t ¢lini¢al siles in the UK and the
USA lo undertake data monitori￿. Th8se visits are instrument81 in building clo$e rel81ions wlh site staff and to ensure
there has been an ass&ssment of the accuracy ol dinical data in the regislry. In addition, we have establi5h8d centtal
data monitoring process85 lo provide a cosl-effectiV6 monitoring of dalg CoM￿etene$s, Iirneliness and consistency.
The INPDR achieved IS09001 accreditation for Quality Management Systems by the 8rit15h Standards In51ilule during
thi5 period for 811 areas related to data collection and management. This accrecfitation dernon5tr8les the INPDR has
robust system for managing the registry and supporting dinical research.
In terms of data use in the INPDR, there have been several approved 5tudie5, r858arch sludE$ and data requests.
These are summar1$6d below.
Research Studlos
St Mary'5 H05Pital M8nchester, UK,"Defining the 58izure burden in Niemann Pick C.
Aston Univ8rsity. UK."Quality ol life (QOLI in patients with Niemann-Pick D1sease Type C INPCI and their carers..
Univer511y College Lordon, Disease piogiession in early and late infantile Niemann-Pick.
University ol Oxford, UK, 'The manif8Stations and rnana9ement ol Inflammatory bNel dise83e in Niemann-Pick
disease type C INPCI patients."
- University ol Oxlord,"Incorpoiation of ASIS into IfE INPDR for NPC cases.
Shared Informatlon
- Genotype reports published for NPC population by Country- NNPDF, Marth 2024.
- Anonymised NPC 8Xtract released to C-Palh, February 2024.
Note review performed on All-slripes database Shared with INPDR to 8s$e$s utility for Us-led Slbling study.
Peer.revlewed Publications
R8eb81. E.M . Wseman, S., Donnelly, C., Malhieson. T. Pounlney. J., Crowe, J. and Hopkin, J., 2024. Real-life impacts
of 011p￿lase alfa.. The experienc8 of pat￿nIs and families taking an enzyme repl8￿ment Iherapy lor acKI
Sphi￿OMY8l1Thase deficiency. Orphan8t Joumal of Rare Diseases. 19111, p.36.
Reports
The Impact of olipudase alfa on adults with ASMD- Part 1 Qu3nlitaliv8 Report. Solomon Mbua, INPOR Justin Hopkin.
NNPDF Toni Mathieson, NPUK Joslyn Crowe, NNPDF Conan Donnelly. INPDR
The impact of treatment with dipudase alfa on adult patients with ASMD - Part 2 Qualitative Repoft Rare Disease
R65earch Partngts.
Page S

INTERNATIONAL NIEMANN-PICK DISEASE
REGISTRY
R8POrt of the Trustees
for the Year Ended 31 March 2024
Achlevement and porforman¢&
Posters
WIORLD COnfgf8n￿, February 2024. Ni8mann-Pick Type C.. An Update by The International Niemann-PicJ( Disease
Registry.
WORLD Conferenc£, F6bruary 2if24, Th8 impacts ol olipudase alfa on adults with ASMD.. palient-reported
experien￿.
- WORLD Conferen￿, February 2024, Real World Data Collection in Niemann-Piek Disease Type C - Data from Early
A¢￿$$ Pragr8m with Arimwbmd.
Communleatlon and engagement
INPDR also wllaborated internationally to undertake research in Niem8nn-Pick diseases. Resutt5 were ￿sented at
national and International meetings Includ1no'.
Niemann-Pid( UK,
The International Niemann-Pick Di88as6s Allian￿.
- WORLD (We'r8 Organising Research on Lysosomal Disease) Symposium. Florida, USA
National Niemann-Pick Disease Foundation Conferen￿, Florida, USA
- Ala ParseghBn Research Conferen￿, Arizona, USA
The iegistry has worked hard to strengthen ils profi18 and Increase inlernalional engagement during th8 last year, in
addition lo pièsenting at international meetiNJs, we established the INPDR Ambassador Programme, where communlty
leaders work to promote re¢ruilm8rit to the registry in their country and to provide 360. feedback lo INPDR with
v￿W to improving INPOR engagemenl and perf0mlan￿.
Flnanclal review
Flnancial posltion
Uniesliicled income in th8 year was £39.143 12023 £109,381) arKI unrestridèd expendilure vas £183,249 12023
£348.3701.
A iestricled giant totalling £81.321 12023 £63,980) w8s awsrded by the University ol Notre Dame. thr¢)ugh its Ara
Parseghian Medical Research Fund and Michael, Marria. arKI Christa Parseghian Endowtnent for Excellence in
Niemann Pick Type C Research. The funding re￿1Ve￿ has en8bled the continuing dev81oprnent ol the Reg151ry.
Alongside the high level of 5UPPOrt and inler8Gtion from the patient and Clin￿al communities. d8mon51rates the potenti81
of the Registry lo make a posrtive diffe￿ nc8 for the global N￿Mann-PiCk patient community. A iestricted giant of
£11.534 was ￿￿1Ved Iiom Sanofi Genzyme loward5 the INPDR forum 2023 8nd the National Lottery Communty Fund
adva[￿ed £19,500 for telling the story of NPDS in the UK.
Deficit on unrestrf¢ted fund
The￿ Is a deficit on the ￿n￿strICted funds. This reflected increased activity and COr￿spOndIng expenditu￿ following an
UnderSper￿ in these funds In P￿vIouS years due to slower activity during COVID.
11 is proposed to address the def￿￿t in the Un￿strICted funds by increasing the contiibutv)ns from INPDR G2Mval'.
Reserves pollcy
There were no fve8 teserves on 31 March 2024 as there was 8 defiat of £7,429 12023 £136,677). The board aim is to
hold six months running costs in feserwe which WOU￿ be £91.624. Further as the registry acb"irities glow a higher ￿Ve1 of
r8serve will be reqviied.
Sustslnablllty & INPDR Gatèway Ltd
A sustainability plan h8$ be8n introduced with th8 aim ol diversifyiro income and providing finanu81 stability. This
includes a commercial subscriplK)n model, a costing miKJel for the non-c(xMmertsal sectors alongsid9 a trust and
corpor8te g18nt strategy.
To support th8 commercial subscription, a commer¢i81 cnmpgny, INPDR Gateway Ltd, was estsblished. An 8PPlOPriata
gtsvernan￿ rn￿e1 was established to allow the Board of Directors ol INPDR Gateway to manage commercial
relaknonships on behalf ol INPOR and ensure that income gener8ted through these activiti85 IS utilised by INPDR to
serve interests of th8 International Ni8mann-Pick community. INPDR Gateway contribuled lo £20,0(YJ 12023
£104,2761 to INPDR.
Page 6

INTERNATIONAL NIEMANN-PICK DISEASE
REGISTRY
Report tsf the Trustees
for the Ygar Ended 31 March 2024
Future plans
The INPDR has devgloped and embe(IcSed a compreh8n3iV8 model for collectiry both rjinician and patient-reported
data, support8d by systems that imprtsve data quality and facilities for clinical and 8pid8miok)gi¢al research. Our
5USt8in8bility p18n, powered by the INPDR G8teway. offers commercial organiz31ions the opportunity tts invest in the
registry, driving growth and broadening th8 servi￿8 we provide.
In the coming year, the INPDR will txjild on Ikese Systems to lurther adv8nc8 our mission, with eonts"nu8d inv8Strnent in
the following key areas".
1. Communication
A variety of resour￿$ will be developed 10 51r8ngthen communication with all INPDR stakehoklers. The registry will
invest in communication expertise and enhancs its presence at nallonal and intemational Conferen￿$. We will 8180
focus on building stronger partne15hips wth the global Niemann-Pick community to promote the INPDR among
clinicians patients.
2. Data Quallty and Cornplfrten855
The INPDR tearn will ¢ollaborale with dinical sit•s woiklwKI8 to improve data COmp￿tene58 and accuracy, ensuring the
gistry effectively Supports a wide range of clinical research iniliatwes. The r8gistry tnanagement team will furth8r
develop this fun¢lion and undertake further initiatives to enhan￿ ￿ntral data tnonitoring pr(Kesses at INPDR.
3. Research Support
In the coming year, the INPDR aims to expand its portfolio 015UPPOrted research studies. In partnership with our
collaborators, we will facilitat8 approved research by offerirKJ clinicians, researchers. and Industry aec8SS to 8nonymized
data and an8lyses. Addbtionally. we will engage in 5pecffic research projects with clinicians 8nd natronal and
intemational patient organEz8tions to deepen the understanding of th8 Niernann-PKk dise8Sè exper￿nc?.
Structure, goyemance and management
Statu5 and Govemlng Document
INPDR is a company limited by guarantee governed by rts Memorandurn and Artides of ASSOCIat￿)n. The cotnp8ny has
ore member, the International Ni8m8nn-Pid< Disease Alliance.
INPDR is also iegistered as a charity with the Charity Comtnk55ion.
Re¢rultm¥nt and appointment of new trustees
Future Tru51ees shall be appointed by th8 Truslees frThr time to lime following 2 nornin8tion ￿￿1Ved Irotn Ihe
Nominations Committee. Trustees ai? VO￿n￿er$ and e8ch takes on responsibilities wthin the organlsatK)n to
co-or(lin8telsupport an aspect of its fundion.
Inductlon and tralnlng of new tn￿10*9
New Trustees are bnefed on their legal obligations, the content5 of memorandum & artidès ol association. the board
ancl dècision tnaking processes. the business p13n and the recent financial performance of the charity. During the
Induction, they are mentored by other Trustees.
Oragnlsatlonal Structur8
The Tiustees are directly responsib16 lor the effeclive goVerTran￿ ol the organis8tion in ac￿rdanCe with statutory
guidance ano legislath)n as provided by the Charity Commissioners and Companies House. The Trustees meet
quarterly with additional contact as requir￿1. in order lo ￿VIeW ièports and lo carry out management and financial
rewews.
Trustees follow a code of conduct. which provides clear guidelines 8$ lo the standards of behaviour, r85pon3ibilities,
and best practi￿ exp8Cted of those involved with the INPOR.
Trusteas ￿￿￿818 the day-lo-day management of th8 organisalion to the Registry Management Team. led by the
Chief Executive Officer, who reports r4ularty to the Board at quarterly virtual meeting5.
Key managemènt remuneration
All trustees give ol their lime IreeFy and no Iiustee received remuneralK)n in the y8ar. The board i8 responsibl8 for
setting employee salary levels. All salaries are b8nchm3rked with similar roles in the volunlary sector, not wth lh8 publ
(K priv8te sectors. The size ol the organisation is also consKJered.
Pag8 7

INTERNATIONAL NIEMANN-PICK DISEASE
REGISTRY
R8POrt ofthe Trustees
for the Year Ended 31 Mareh 2024
Stru¢ture, governance and management
WSder nelwork
The eh8rity's member is the Internation81 Niemann-Pick Di58a8e Allian￿ IINPDAI which is an alliance ol non-profit
support oiganisations who ar8 associaled Mrith Ihe rare group of genetic diseases known collectively 88 Niemann-Pick
Diseases. The Alliance provides a forum where, throuuh the exchange ol inlormatK)n, experien￿ and knth￿edge.
global progress can be accelerated
Rlsk management
As part of the Quality Management System, the INPDR rnaintains a risks and issues log. Risks are managed on 8n
orooing basts by the R￿iStry Management Team and reviewed by Board Mernbers 81 a quarterly Man8getnenl Review
Meeting.
The registry OP8rates in a highly regulated dinical research environment arvj musl ensurè ￿mplianCe vrilh data
protection law and good ¢lini¢al practice. INPDR operale5 In compliance with the European Medicine Agency IEMAI
Guidelinès on the Management of Patient Registries and the ieuistry has reC￿Ved a letter ol support trom EMA as g
resource for dinical res8arch.
To manage ii5k5 ￿lated to data protection and ensuie the privacy of ￿gistry partlC4P8nts. INPDR employs the servi
ol a ¢on$ultant Oala Prote¢tK)n offi￿r. In addition. durino the year 2022123, our tschnology partner, OpenApp was
awarded IS027001 acciedilalion for data se¢urity, provKling further Confiden￿ that the INPDR are well-placed lo
en8uie patient privacy
th regard lo data protection, there were no adverse events, incidents or breaches reported during 2ry23124 and no
data $ubj8ct ligh15 Nuesls.
Recognising the contribution of voluntsers
We a￿ extremely grateful to those who give their tirne as volunteers in su5WQrt of the INPDR. Without Iheii d8dicated
and continued 5UPPOrt, we woukj not be gb￿ lo achieve 811 that is ￿quired on behsll ol our oryanisstion and community
statement of tnJ8tètrs' respon8ibilitles
The tru51e8s (who are also the directors of Inlemational Niem8nn-Pick Dkqease Registry lor the purposes ol company
wl are r8sponsible for preparing the Report of the Trustees th8 financial slalements in accordance wrth applicab
law and Unit8d Kingdom Accounting Standards (United Kingdom Genet81ty A￿pted Accounting Practi￿1.
Company law requiie5 the trustee5 to prepare financial statements lor each financial year which give a true and fair view
of the 5tste of affairs ol the tharitsb￿ company and of the incoming resource5 and application of osources, including
th8 income and expenditure, ol the th8rltsb￿ company for that pefiod. In prepa￿ng those finan￿81 statements, the
trustees are required to
select suitable accounting pdicies and then apply them consistently.,
obseNe the methods and principles in the Ch8rity SORP",
makejudg8ments and estimates that are ￿aSOnable and PDJdent'.
prepare the finanoal statements on the going concem basis unless il is inappropriate to piosume that the charitable
company will continue In business.
The trustees are responsible for keeping proper accounting records which disclose with reasonable accuracy at any
time the Iinancial position of the eharilable company 2nd lo enabl8 thèm to ensure that thè financial st8lemen15 Comply
with the Companies Act 2006. They a￿ also responsible lor S8f￿￿&[dIng the assets of Ihe chantabb (x>mpany and
hen￿ f¢w taking reasonable step5 ft)rthe prevention 8nd detection of fraud other irregul8ritie5.
This report has been prepared in 8Cwrdan￿ with th8 swal provisions of Part 15 01 the Companles Act 2￿8 r81ating
to srnall companies.
Approved by order of the board ol trust8es on 24 December 2024 arKI signed on I￿ behalf by..
J E Green- Tiusteè
Page 8

Indèpendent Examlner's Report to the Tru8tees of
International Niemann.Pick Dis8ase
Reglstry
Independent examlnerfs report to the trustees of International Nlemann.Pick Disease Registry I'the Company'l
l ￿port to the charity trustees on my examination ol the accounts ol the Company for the year ended 31 Maich 2024.
Responsibi15ties and basls of report
As Ihg charity's trustees of th8 Company land alsts its directors for the purposes ol company lawl you are responsible for
Ihe preparation ol the account5 in arordanTr with th8 requirements ol the Compan￿5 Act 20061'the 20W Act'l.
Having satisfied mysew that accounts ol the Company are not required to be audited urKJer Part 16 of the 2CO6 Acl
and are eligible for independent examination. I report in respect DI tny examination of your charity's accounts as carried
out undei Se¢lion 145 01 the Chaiitie5 Act 2011 I'the 2011 Acl'l. In carryin9 out my examinat￿￿ I have followed the
Directions given by the Charity Comrni5siDn under Seclion 145151 Ibl of the 2011 Act.
Independent examinels stalemtrnt
I have completed my 8x8minalion. I confim that no matters have come to my 8ttenty.on in COn￿¢(lon with the
examination giving me cause to believe..
aceounling records were not kept in r8spect ol the Company a5 required by Section 386 olthe 2006Act', or
the 8ccounls do not accoid with Ihose records," or
the a¢counls do not compw with the accoUn￿ng requirements ol Secknon 396 of the 2006 Act other than any
reqU1￿ment that the accounts give a tIU8 and f8ir view which is not a mattor consider￿1 as part of an
independenl examination.. or
the accoun15 have not been p￿Pared In accordance with the tnelhods and principles ol the Statement of
Recommended Pra¢U'ce for accounting and repo￿n9 by charibe8 (applicab￿ to charities preparing their
account5 in gccordance with the Financial Reporting Standard applicable in the UK and Republic of Ireland IFRS
10211.
I have no concerns and have come across no other ma118r5 in connection wilh the examination to which attention should
be drawn in this report in ord81 to engbEe a proper understanding of the accounts to be reached.
Christopher T Spalding
James Ancserson & Co
Charterecl Accountants
Ptrntland E5tste
Str&iton
Edinbuigh
EH20 9QH
24 De￿mber 2024
Pag8 9

INTERNATIONAL NIEMANN-PICK DISEASE
REGISTRY
ststement of Financlal Actlvltl85
Ilncorporatlng an Income and Exp8ndlture Accounti
for Ihe Year Ended 31 March 2024
2024
Totsl
funds
2023
Totsl
funds
Unre8tricted
fund
Restricted
fund¥
Noteg
Income and endowments from
Donations 8nd ￿0a¢re$
520
92,355
92,875
69,085
Charitable actlvltles
Charitabk acliwties
38,623
38,623
104,276
Total
39,143
92.355
131,498
173,361
Expenditure on
Charitable actlvltles
charitab￿ activities
183.249
124,437
307,686
415.907
NET INCOMEIIEXPENDITUREI
1144.1061
132.0821
1176,1881
1242,5461
Reconciliation of ￿ndS
Total funds brought fotward
136,677
50,898
187,575
430,121
Total funds ¢ar¥ied forward
17,4291
18,816
11,387
187,575
Th8 notes form part ol these financial statements
P8ge 10

INTERNATIONAL NIEMANN-PICK DISEASE
REGISTRY
Balance Sheet
31 March 2024
2024
2023
Notes
Flxed assets
Inveslmenls
Current a¥sets
Deblo
Cash at bank
10
22
25,363
90.056
116.772
25,385
2[￿,828
Crgditors
Amounts falliro due Within on8 year
113,9991
119.2541
Net Current assots
11,386
187.574
Total ass8ts lass current Ilabllltles
11.387
187,575
NET ASSETS
11,387
187,575
Funds
Uni881iicled fijnds
Restricted ￿ndS
13
17,4291
18,816
136,677
50,898
Totsl funds
11.387
187.575
The charitablè company is entil￿d to exemptron from aLKJit under Section 477 of the CoM￿nieS Act 20C6 for the year
ended 31 Mareh 2024.
Th8 mgrnbets have not ￿quired the company lo obtain an audit ol ils financial Statemen￿ for the year ended
31 March 2024 in accordano wrth Section 476 of the Compani&s Act 2006.
TIE Iruslees acknowledge their responsibilrties for
ensuring that th8 Charitsb￿ company keeps accounting records thai 1X)mp￿ wrth Sections 386 and 387 01 the
Companie5 Act 2006 and
preparing financial statements which gwe a true and fair view of th8 stale of affairs of the charitab￿ company as
st th8 end of e8ch fina￿la1 year arKI of ils surplus or deficrt for each financial year In accordan￿ with the
requirements ol Sections 394 and 395 and which othetwise comply with the requirements of the Cornpanies Act
2006 relating to financi81 statetnents. so lar as appllcable to th8 charitatde company.
These financial slatetnents have been prepared in accordan￿ with the provisK)ns applic8ble lo charitable compani8S
subject lo the sm811 cotnp8nie5 r8gime.
The financlsl 5ta18m8nts We￿ approved by the Board ol Trustees and 8UtFK>fi8ed for issue on 24 De￿rnb8r 2024 and
were signed on its behalf by..
J E Green- Trustee
T1￿ notes fom part ol thès8 financial statements
Page11

INTERNATIONAL NIEMANN-PICK DISEASE
REGISTRY
Notes to the Financigl Statements
forth& Year Ended 31 Mar¢h 2024
Accounting pollcies
Ba$ls of preparing the financial ststements
The financial slat8m8nts ol the ¢h8ri1ab￿ company, which is a public benefit enb.ty under FRS 102. have been
prepared in ac¢ord8nce wrth the Charities SORP IFRS 1021 'Accounling and Reporting by Charities.. Staletnent
of R8commended Practice applicable lo charitie5 P￿parIng their accounts in accordan￿ wrth the Financial
Reporting Standard applicable In the UK and R8public of Ireland IFRS 1021 leffe¢live 1 January 20191,. Financial
Reporting Stand3rd 102 'The Finanoal Reporting Standard applicable in the UK and Republic of Ireland, and the
Companies Act 2006. The financial Statements hav8 been prepared urKJer the historical cost convention, wrth the
&X￿ptIOn ol investtnents which are included at market walu8.
In18mational Nianann-Pick Disease Regk3try meets the definilion of a PLblic benefit entity undèr FRS 102.
Income
All in¢L¥ne is recognised ￿¢8 the charity has enti118ment to the incomg. there i% suffKient cert8inty of re￿Ipt
and so it is probable that the in(x>m8 wll be ￿￿1ved. arKI tha amount of incotne re￿Nable c8n measured
Donations and grants are recognised when they have been communicated In vrriling with notification of both the
amount and settlernenl dal8 In the event that 8 donation or grant is subject to conditions that require 8 level of
performance befo￿ the ¢h8rity is entitled to th8 funds. the income is d8ferred and nol recogniseis until either
those corKlilions are fully met, or th8 fijifilment of Ih05e conditions is wholty within the control of the charity and il
is probable that those conditions will be lulfilled in the reporting period.
ExpendIt￿re
Expenditure is recognis8d as soon as th8re is a legal or constiudive oblualion c0tntnitt1￿ the charity to that
expenditure, il is probabl8 that a transfer of economic benefits wll be reqUI￿d in sett18m&nt and the amount of
Obl￿atIon Can be measured Trliably.
All eXper￿lture is accounted for on an accruals b8sis. All expenses, including support ¢0515 gtsvernan
costs, are alloca18d or apportioned to the applicable expenditurt headings in the Statement ol fin8nci81 acuvities.
ExpetKfiture on ¢harilable aclivib'es includes any VAT which txnnot be recovered and is reported a5 part of the
expenditure to which it relates and compri58S those Costs incurred by the Charity in the delivery ol ils 8CtMties
and services for its benefickanes. It includes both costs that can be allocated diTect1y to such actwities and those
costs ol an Indiffjct nature ne[￿Ssary lo support them.
Taxatlon
The tharty is exemptfrom corporation tax on its charitabb activitiès.
Fund ac¢ountÈng
Unrestricted lurKls can be used in accordance wrth the charitable objectwes at the discretion of the trustees.
Reslricled fund5 can only be used foi particular restricted purposes within the objects ol the charity. RestriclDns
ar￿e when specified by the donoi 01 when lunds are ra￿$ lor particular r8Stricted pury)oses.
Further ex￿anatIOn aft￿ nature and purpose ol each fund is induded in the notes to the finanual ststetnen
P•nslon cost$
The company operates a defined eontribulion $ch8me, the assels of which are hdd separatety from those of the
charity Th8 PenS￿n cost ¢harge represents Contributions payab￿ by the charity to the schemg.
Investments
The investment in the group undertaking Is measured at cost less impairment on the basis that it repiB5ernts
share5 In an entity Ihat is not publidy traded arKJ the lair value cannot othemise be rne8sured
r81iably.
Debtors and creditors recelvable I payable wlthln ono yoar
Debtors ancl creditors with no stated interest rate arvj re￿1V￿ble or payable wrythin one year are re¢ord8d at
transaction pri￿. Any1085es 8rising from impairtnenl are reco9nised In expenditure.
Ca8h in bank and In hand
Cash at bank and cash in hand includes cash and short term highly IiquKI invèstments.
Page 12
(x>nkn'nued...

INTERNATIONAL NIEMANN-PICK DISEASE
REGISTRY
Notes to the Financial Statements contlnu8d
for the Year Ended 31 Maich 2024
A¢¢ountlng pollcles- continued
Golng concern
Th8 financi81 statements have been p￿pared on 8 going Contsrn basis as the trustees bal￿v8 that no material
uncertainties exi51. The company has a defi￿8￿cY of as*ts on ils unrestricled fvnd. In response plans have
been drawn up to address this. The trustees have considered t1￿ level of funds helij and the expected 18v81 of
income arKI expe[￿llUre for 12 months from 8ulhorising thes8 financial statement5. The budgeted income and
expendilurE 15 sufficient wth the level of ieseNes lor the charity to be able lo conlnue as a going contsm.
Donations and legacles
2024
Total
funds
2023
Total
funds
Unrestricted
funds
Restrlcted
funds
Donations
Grants
520
520
92.355
5,105
63.980
92.355
520
92,355
92,875
69.085
Grants re￿1Ve(l, Pr￿uded in the above, are as follows..
2024
2023
Sanofi Genzyme
University of Nolre Dame du Lac
National Lottery Community Fund
11.534
61,321
19,500
63,980
92,355
63.980
Income from charitab18 a¢tivities
2024
2023
Activity
charitab￿ actiwtie5
Contribution lo Research
Recharge to INPDR Gateway
Ltd
Third party reirnbutsernenl
18,459
20,og2
ChaiitalAe 8cli¥iti8s
Charitable 8cliwties
20,￿)0
164
84.184
38.623
104,276
Charitable activities costs
Direct
Costs Isee
note 5
Charrtable activiti8
307.686
Page 13
continued...

INTERNATIONAL NIEMANN-PICK DISEASE
REGISTRY
Notes to the Flnancial Statements. continuetl
for the Year Ended 31 March 2024
Direct costs of charltsble actlvltles
2024
2023
Staff costs
Equipment costs
Registry hosting
Travel & meeting5
Website ¢o$ls
other staff costs
Offi(E expenses
Promolional expenses
Executive rneetings
Independent exatninels fee
other regulatory eosts
Consultan¢y
Legal fees
Rebrgnding
Research
HIC Maint8nan￿ & support
129,563
103,305
1,299
47.913
38.583
1,070
689
8,184
158
2.426
852
60.001
89,619
23,572
9,024
29,212
21,740
29.722
1.027
9,520
5,826
2,424
1.974
3.713
71.669
20,068
10,440
307,6e6
415,907
Tru$tee$' remuneratlon and benefits
There were no trustees, remuneration or other benefits for the period ended 31 March 202412023- nil)
Trustees. expenses
During the period one trustee was wrnbutsed £ 58312023 £1031 for travel exp8nses.12023- 11
staff costs
2024
2023
Wages and salaii85
Social security costs
Oth8r pension costs
121,223
S.993
2.347
97.527
3,897
1,881
129,563
103,305
The average monthly number of employees during Ihe year was as fdlows..
2024
2023
Coordination and 8dministration
Registry Manager
The Chief Executs"ve OffKer is k8y Man4emeDt and his total ernployee benefits lindud1￿ Nl and Pension) ￿re
£65,455. 12023 £53,915)
Page 14
continued...

INTERNAfioNAL NIEMANN-PICK DISEASE
REGISTRY
Notes to the Financial Statements- continued
for the Y8ar Ended 31 March 2024
Comparatlvès for the statoment of financlal adlvitles
Unrestrlctgd
fund
R8Stricted
funds
Totsl
funds
Income and endowments from
Donations and legarjes
5,105
63,980
69,085
Charltsble activities
Chaiitable activities
104,276
104,276
Total
109,381
63,980
173,361
Expènditure on
Charitable actlvltles
Charitable acb¥rylies
348.370
67,537
415,￿7
NET INCOMEIIEXPENDITUREI
1238,9891
13,5571
1242,5461
Reconclllatlon of funds
Total lunds brought forward
375,6e6
54.455
430.121
Total fund8 Carried forward
136.677
9).898
187,575
FIx8d asset Investmonts
shares In
group
undertakings
Markèt value
Al 1 Apri12023 and 31 March 2024
Nèt book value
Al 31 Msrch 2024
At 31 March 2023
Ther8 were no investment assots outside the UK.
Th8 companls inveslrn8nts al the balance sheet d8te in share capital of companies include the following..
INPDR Gateway Ltd
Registered office.. United Kingdom 12706306
Nature ol busin8ss'. Data provider lor research
Clas$ of share..
holdlng
100
31.3.24
31.3.23
Aggr8gate capital and reseN8s
(Lossllprofit lor the yebr
149,4461
152,1601
2,715
2.714
Page 15
continued...

INTERNATIONAL NIEMANN-PICK DISEASE
REGISTRY
Not&s to the Financial Statements- contlnued
for the Year Ended 31 March 2024
10.
Debtors.. amounts falllng due within one year
2024
2023
Trade debtors
Prepayments
22
84.184
5,872
22
J.056
11.
Creditors: amounts falling due wlthin one year
2024
2023
Srtial security and other taxes
Accruals and deferred income
2.854
11.145
2,532
16,722
13,￿9
19,254
12.
Analysls of net assets befv￿en lund8
2024
Total
fvnd8
2023
Total
funds
Unrestrlcted
fund
R&strS¢ted
fvnds
Inveslm8nts
Curienl assets
Current liabilities
6,569
113,9991
18,816
25,385
113,9991
206.828
119,2541
17,4291
18,816
11,387
187,575
13.
Movement In funds
Net
mov6ment
in funds
At
3113124
At 114123
Unrestrl¢t¢d funds
Ge￿ra[ fund
136,677
1144.1(bl
17,4291
Restricted funds
APMRF
National Lottery Community Fund
.898
150.3641
18.282
534
18,282
50,898
132,0821
18.816
TOTAL FUNDS
187,575
1176,1881
11,387
Pag8 16
continued...

INTERNATIONAL NIEMANN-PICK DISEASE
REGISTRY
Notes to the Financial Stat0wrtents- contlnuèd
for the Year End8d 31 March 2024
13.
Movement In funds - contfjnued
Net mov8mgnt in funds. included in the ebove are a5 follow$'.
Incomlng
r&$ources
Resouices
expended
Movement
In funds
Unrestrlcted funds
General fvnd
39,143
{183.2491
1144,1061
Rgstrlcted fund8
APMRF
Sanofi
Nstional Lottery Cornmunity Fund
61,321
11.534
19,500
1111,6851
111,5341
I￿,384)
18,282
92,355
1124.4371
132,0821
TOTAL FUNDS
131.498
1307,6861
1176.1881
Comparatlves for movement In funds
Ntrt
movement
in funds
At
3113123
At 114122
Unrestricted funds
General fund
375,666
1238,9891
136.677
Rèstrlcted funds
APMRF
54.455
13,5571
50,898
TOTAL FUNDS
430,121
1242,5461
187.575
omparatsve nel tnovement in funds. inc4ud8d in the above ar8 as follows..
Incomlng
resouree8
Resource$
eXP8nded
Movement
In funds
Unrestilcted fund8
General fund
109,381
1348,3701
1238,9891
Restricted funds
APMRF
63,980
167,5371
13,5571
TOTAL FUNDS
173,361
1415,9071
1242,5461
The general fund is ftsa lo use in accordan￿ with the objects olthe charity.
The APMRF fund is to accelerate prc¥ress by facilitating iegislry recruitment in the USA.
The Sanofi furKI w8S towards the INPDR 2023 Forurn
The National Lottery Community Fund is lor telling the story ol Niem?nn-P￿k Dis&ases in thè UK
Page17
continued...

INTERNATIONAL NIEMANN-PICK DISEASE
REGISTRY
Notes to the Flnancial Statements- contlnued
for thp Year Ended 3q March 2024
14.
Relatsd party dlsclosure
Tha International Niemann-Pick Disease Alliance IINPDAI is the only memb8r ol the company. Duriro the year
costs were charged by INPDA to INPDR of £3.014 12023 £2,920). INPDA paid shared costs of £4,422 12023
£5781. At 31 Marth 2024 there was a balance due to INPDA ol £Nil 12023 £5781.
Niemann-Pick UK INPUKI 15 a member of SNPDR. During the year there were rechaiges for sha￿d staff,
travel and oveiheads etc by NPUK lo INPDR of £27,142 12023 £20.9911. INPDR received inwre in the year
from NPUK of £7,981 12023 - £10,89gi. Al 31 March 2024 there was a balance due to NPUK of £2.650 12023
The INPDR (Registry) fully owns a company, INPDR Gateway Ltd (Gatewayl. which carries out the comrneicial
operations of the R￿lsIry. Ouring the year 2 sum ol £Nil12023 £5.0001 was ￿nI by Registry lo the Gateway.
In addilion iecharges and setvice charges weie made to the Gateway in the year of £20,761 12023- £84,184>. A
sutn of £22 was due from the Registry at 31 March 202412023 due to £84.1841.
Page 18

INTERNATIONAL NIEMANN.PI¢K DISEASE
REGISTRY
Detailed Ststement of Financlal Activitles
lor the Year Endad 31 Maich 2024
2024
Total
funds
2023
Total
funds
Unrestricted
funds
R8Strlcted
fund5
Income and endowments
Donallons and legacies
Donab"ons
Gr8nts
520
520
5.105
92,355
520
92,355
92,875
Charltablg actlvltlè8
Contribution lo Research
Recharge to INPDR Gateway Ltd
Third paty reimbursernent
18.459
20,0
164
18,459
20,000
164
20.092
84.184
38,623
38,623
104,276
Total Incomlng r8sour¢es
39,143
92,355
131,498
173.361
Expenditure
Charitable actlvltles
W8ges
Social security
Employer pEn5ion
Equipment costs
Registry hosting
Travel & meetings
Website costs
Olhgr staff costs
Office expenses
Promots"onal exp8nses
Executive meetings
Independenl examinerts lea
Other iegu18tory costs
Consultancy
Legal fees
Rebranding
Resear¢h
HIC mainlenarice & support
74.666
5.993
2.347
46.557
121.223
5.993
2.347
97,527
3,8g7
1.881
1.299
47.913
38,583
1,070
21,740
11.921
1,027
21,740
29,722
1,027
17.801
9,520
5.826
2,424
1,974
3,713
11.590
9,520
5.826
2.424
1,974
3,713
71,669
8,184
158
2,426
852
60.001
89.619
23,512
9,024
29,212
60.079
20,th8
10,440
20,068
10,440
183,249
124,437
307,686
415.907
Total rèsources eXp￿ded
183.249
124.437
307.686
415,907
Net loxpendlturglllncome
I144.1￿)
132.0821
1176, 1881
1242.5461
This page does Th)t forrn part of the statutory finan(xal staten18nl
Page 19