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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 Trustees and
Flnanclal Ststements for th8 Year Ended 31 March 2025

INTERNATIONAL NIEMANN-PICK DISEASE
REGISTRY
Contents of the Financlal Statements
for the Year Ended 31 March 2025
Pag
Ref8rgnce and Adminlstratlve Detalls
Report of the TNstee5
2 to 10
Independ&nt Examlnerfs Report
statoment of FÉnanclal Activiti8s
12
Balance Sh091
13
Noles to the Financlal Statemonts
14 to 19

INTERNATIONAL NIEMANN-PICK DISEASE
REGISTRY
Relèrenco and Admlnlstrative Detalls
for the Year Endèd 31 March 2025
Trusteos
J E Green
T HNvol
Mrs T A Malhieson
Dr A Dardis
Dr M Vanier
Company secretary
Mrs T A Mathieson
Registered officè
Suite 2e
North Sands BUSI￿58 Centre
Lib8rty Way
Sunderland
Tyne and Weal
SR6 OQA
Reg18tered company number
10646129 (Eryland and Ws1è51
Reglster8d charlty numb8r
1175311
Independent examlner
James Anderson & Co
Chart8red Acctyjntsnts
Pentland Estate
Slrailon
Edinburgh
EH20 9Q
Chref executiv8 officer
Conan Donn811y
Pag8 1

INTERNATIONAL NIEMANN-PICK DISEASE
REGISTRY (REGISTERED NUMBER.. 106461291
Report oflhe Trust8es
for thè Year Ended 31 March 2026
trustees who are alsD director5 01 the Charity for the purposes ol th8 Companies Act 2006, PT8sent their report with
the financi81 statements of the thatily for the y88r ended 31 March 2025. The Irust8es have adop18d the provisions 01
Accounting and Reporting by Charitses." Statement of Recomtnended Practi￿ applicable to charities preparing their
accounts in accordan￿ with the Financi81 Reporting Standard applicable in the UK and R8public of Ireland IFFiS 1021
18ffeclive 1 January 20191.
Objectives and activltles
Background
The InlernatK>nal Miemann-P￿k Disease Registry IINPDRI is an indep8ndent, nol-foi-profil charitable 0￿panY
Iste￿d in Ihe UK. Cre8t8d by a collaboration of patients, health professionals and medical researchers, il offers a
sing￿, diseastrspeafic global ￿gistry data resour￿ that Indudes both dinical data and pat￿nt reported data lor Acid
sphing0m￿linase DeficierKy IASMDI NiemaTrn-Pick dise85e, and Niernann-Pick disease type C.
Due to its r8rity, registr8tion for Niemann-Pick diseases is particularly important. Registrab"on can prowde sufficient data
to understand its ¢omp18x natural history, support aC￿lerated reciuilment to trials and facilitate Ihe d8vebpment and
monitDring of care Sta￿ardS.
Mission, Vlsion and Principles
The mission ol the INPOR L% to document the Niemann-Pick patient expeiience,. lo irnprove diaonos15, treatment and
a￿, to advdncè rtsearch and ultimately. to improve health oulcnrne5 lor all those affected.
Our vision is the C￿atIon and ongoing devebpment of a comprehensve. int8mational data resource. specific lo
Niemann-Pick dis88ses which benefits pat￿￿1$ by in(xeasiro understanding of these rare condits"ons, encourages
ettiuent aTKI timely diagnosis, 8n8b￿S piogiess in research and dinical trials. and facilitates the developrn6nt of
therapeutic interventions.
INPDR advancès our ViSK)n by adhering to the following prinCi￿eS.
o W8 iespect arKI ptote¢l pa118nt privacy, 8nd build trust among our pati8nl community.
o We strive for excgll&nce through eontinuous Ynprovernent. piofessh)n81ism and expgrt 8UPPOrt.
o We operate to high8St levels ol s(i6ntifi¢ int89rity and promote high-quality research.
o We are transparent in communicati￿S take respon5ibilty for our actions and deagons.
o W8 foster an environment of collaboration to improve outcomes in Niemann-Pick diseases.
o We embffdce innov8tion and change as appropriate to our n8eds.
We shall ietnain independenl of 8dvocacy, policy and commercial interests and let data tell the story ol
Niemann-Pi¢rt diseases.
A key strerolh of lh6 INPDR is the wlaborative rdationships developed from the outset and enabling the powerful
combination ol patient leadership and piof6ssional implemenl81ion. This ensuras the bgitimacy of th8 patpnl
perspedive and ensur8s that il Is 8t the f￿art of ourorganisation and mission.
Page 2

INTERNATIONAL NIEMANN-PICK DISEASE
REGISTRY (REGISTERED NUMBER.. 106461291
Report ofth8 Trustees
for the Year Ended 31 March 2025
The INPDR will work to enhance and laclSrtalg prO9￿SS through the following aims and objetlives..
Alms
o To build a high quality, cOMp￿te Inlemational populatv)n-based Nierllann-P￿k D￿ease Registry
o Protect patient privacy arTrd ensure ￿MplIanCe with data protection and the highest dinical stsndards
o To facilitate clinical research through dala sharing and provision ol analytical sewices
o Provide eVKIen￿ to faeilitat8 advocaDJ and inform public debate
Oble¢tives
o To de5crib& tr* epK18mioicK)y and natur81 history ofASMD Nbm8nn-Pid( disease and Niem8nn-Pidr Type C
o To support development o15tand8rds ol diagrTrosis. clinical management and qu81ily ol ca
o To establish genotype-phenotype correlats.ons
o To Pfovide support for equal access to biochemical and g8netic testing
o To facililale a¢celeraled r8cruilmenl lo intetV8nlional studigs
o To pro￿de a regulatory compliant registry to salisfy p05t-marketirKJ obligats'or
o To 5UPPOrt regulztory interaction betW8en natM)nallsuiKanational medi¢ines agencie5 and pharrnac8Utical compan￿5
o To accelerate progi&ss in undetstanding NPD, by encouraging educalion DI health professionals and researchers
The INPDR developerj arKI maintsins a r￿UlatOry-¢oMpl1ant, secure dat8b8se of consenting patien15, Wlth appropr￿te
se¢urily, management, and controls. A Secu￿ web-b8sed electronic d8ta capture IEDCI plalloim is used for the
systematic arKI unifom collection of clini¢al data, p8tient-reported data and cAregiver-reported data ol patients
diagnosed wth either ACKI Sphlngomyelinase Deficiency IASMDI or Niernann-Pi¢k Type C INPCI. The 8clive
parbcipalion of clinicians, patients and caregivers from around the world f8cilitales the collection ol detnographio
informat￿n, d18gnoslic results, treatment, atKI health outcomes data. The INPDR is a k)ng-lerm in4￿"atIve that wll
conl'nue to glow arn* to create methanpsms to ensure that the data Collecleil wod(s hard6St to serve patient needs.
Signlfl¢anl actTrvltles
In the last year we worked to continue improving INPDR'S impad by increasing global ￿¢rUItM8nl. supporting data
collection. enhanong data quality activities and fa￿ll￿ting and supporting meaningfijl research. All activities undertaken
in the reporting p8ri0d moveil the charity foNard in its mission to advan￿ r8search and impiove heamh oirtcomes in
Niemann-Pick (Sis8ases.
Cllnical Data Collection
24 medical contw were Included in the Clinicpn Reported Database ICRDI lo collect clinical data frorn 488 patients in
2024125, consisting 01120 palients with ASMD and 368 with NPCI. As well as maintainin9 collabofab.ons with ￿ntr8S in
North America, Soutth Arner￿a and Europe, new centres in Ihe Middl8 Easl and Br8ziI initiated data collection. 46 new
sites have been £ppio8ched lo collaboiate. with di5CUSsions in progre88.
Patient Reported Data
The Patient Reported Database IPRDI had 8 total of 158 pat￿nts and r8regwers enrolled in thp 2024125 pariod.
Questionnair8 responses were sought trom p8rticipant5. with 83 quality of Pilè surveys and 101 symptom. severity and
unmet need assessment r8turns obtained. Behveon Dec8mber 2024 and March 2025. direct promotion of the PRD w8S
pau58d to supwrt a UK patient organisalion rese8rch inilialive, a comprehensiv6 Patient and car8giver surv8y Isee
'Rèal Wortd EXper￿nCe ol NEmann-Pick disease Type C Patients and ca￿giVe[s, below). The decision wa5 made to
encour8ge participation in the research initiative. The survey pmject supported the PRD by co118ting contact details ol
pattiupanls who w8re Inte￿$ted in joining the PRO.
The PRD i% currently in English. but 8 multi-18nguage solution is in development for 2025. Dats quality monitoring was
strengtfEned in the US and UK. supported by the Ara Pars8ghi8n Medical Research Fund, with vi&ts to clinical sile5 to
ensure data accuracy. INPDR also achieved Is0￿)1 8c(Thdilalion foi Qu81ty Management Systems. eonfirmiw 115
strong registry aThY researth support capabilities.
Page 3

INTERNATIONAL NIEMANN-PICK DISEASE
REGISTRY IREGISTERED NUMBER= 106461291
Report of the Trustees
lor the Year Ended 31 March 2025
Oble¢tlves and actlvltlos
Anonymised InfoTmallon sharlng
To meet the a￿￿ of laalilating dinlcal research through data shallng, INPDR has worked with researc￿r$ to submit
requests lor anonymised registry data. Intemational applicants of differing areas of 8xpertige and backgrounds have
proposEd research piojeds which explo￿￿ differing aspects of Niemann-Pick, includiThJ diseas8 mechanism5,
movement disorders. ￿tIent and caregivei reported expel￿n￿$. and disease management and progression.
Publications, Reports and Posters
INPDR was involved in reports and publications induding, a 2024 publication in Orphanet Joumal of RaE Diseases that
exploi8d the rea1-life impa¢ts of olipudase alla on ASMD pat￿￿15 their famil￿s. Continuing work previously
undertaken, INPDR published a second quanlilalive and qualitative report on the impact of olipudase alfa on adults with
ASMD. wth contributions from the INPDA. NNPOF aThJ NPUK.
INPDR also produ￿d and Contributed to sever81 conference presentations, including at the annual WORLD Symposium
in Febru3ry 2025, where thre8 P05ter abslr8Cts were acc8pl8d and presented. Additionally. pr85entations were
dglivered to multipkg patient cornmunily meetings in Europe, North America and Australia to disserninate research
finding5 8nd proV￿e audien￿ updates of INPDR progr8S8.
Public bet)efSt
In shaping our obi'eclives for the year and planning our activities. the trustees have [X>nS￿ered th8 Charity
Commission'5 guidan￿ on public benefit, including the guidanc8'publL benefit.. running a charity IPB21'.
Achlevement5 and perfomiancè
Charltable actlvltl85
Funding in the form of unrestricted eduratlonal grants from Industry stakeholders enabled the charity to carry oul its
activities. Ill 8ddith)n, INPDR re￿iVed reimbursement for the Servi￿ it prowded to the INPDR Galeway.. Further
lunding came from the Ara Par5eghian Medical Rèsearch Fund the National Lottery Comn)unity Fund.
We have continued to maintain a strong presence in the field of research and hav8 collaborated on a global b8sis wth
olh8r iart disease organisations, reseèreh and academic institutions, regulators. and industry to progress and further
develop the registry, and to facilitate re58arth and clinical devdopmenl.
We conlinue lo sustain appropriat& relationship5 wlh the pham)aceulic81 industry. particularly those engaged in clini¢a
programmes and aclNities in the field of Niernann-Pi¢k diseas85 and to work c105ely with the glob81 patient advocacy
leaders supportir¥J the Niem8nn-Pick community. This Includ88 the International Niemann-Pi¢k Di5885e Alliance. whose
represenlatwes ale appointed to th@ INPDR Board of Tiuslees and Scientific Advisory Committee.
We conb.nued to Wlth the global Niemann-Pick community through virtual and in-pEr50n confeientss and focus
groups.
Pa98 4

INTERNATIONAL NIEMANN-PICK DISEASE
REGISTRY IREGisfERED NUMBER.. 106461291
Report of tho Trustees
for the Year Ended 31 March 2025
Achievement5 and performance
Durlng the 2024125 reporting peri1￿. we have worked to St￿ngthen INPDR'S impact and foster positi￿ progress in
NÈm8nn-Pick research through the following achievements and performance..
-The number of participating clinieal siies increased to 24 across EuroFe, North Arnerica and South America. Through
INPDR'S Collaboration with tfEsts sites, 488 patient rec(Kds were co1￿cted sin(x the laU￿h of th8 CRD. formed of 120
ASMD and 368 NPC rewrds.
- 46 new dinical siles from across the globe, including Eurote, North Atnerica. Soulh America. Australia, ASH and Afllca
engaged with INPDR to initiate a collaborstion lo collect patient records. Thi5 laid the foundation foi Signif￿ant
expansion ol the INPDR'S footprint. enabling iepiesenlation frotn r8gions undersèrwerj by clinical research opportunities.
The Patient Reported Database g￿W to include 158 patients and Ca￿gIve[s. with an a¢￿ptable numlEr of
questionnaire and survey responses obt8ined. A temporary hall on PRD promotion was imposed between December
2024 and March 2025 to support a p8tient organisalK)n research inrtialive. which may have impacted on ￿¢rUitment
rates 3rKI sutvey responsès. A5 th8 research Initiative was desvJned to collect contact details of pariicipants
interested in the PRD. we 3ntlUP8te this will result in fuluie PRD partitipanl iecruilrnenl.
il& th8 PRD is currently available in Eng115h language, wotk has progressed 5ignific8nWy wth technology
partn8r. OpenApp, to develop a mu4￿.ple-raTrYUage solution with the exp8clalion that this will be rolled out in 2026.
A key u￿ale lo the PRD was the introduction of lunclionallty allowing caregiver participants to indicate their lov￿ or
have pgssed away Sin￿ enrolment. This ends the caregiverfs study partiapation and prevents potanlially distressing
ernails requesting new surfftys lo be completed from LEing 58nt to the Ca￿91ver.
During the reporting period, INPDR'S Clinical Research Associates ICRAI undert(M)k several vists of CRD clinical siles
to undertak8 data quality exercises. thereby 5UPPOrting the accuracy and completeness of collected data. These visits
a150 provided an opportunity to maintain and str8nglhen relationships with personnel at the dinical siles and allows for
fèedback and engagement from one ol INPDR'S key collaljorator s&clor.
Addilion8lly. INPDR's CRA'S supportèd lh8 develO￿ent of cor8 data quality prO￿SseS. with input from INPDR'S ChEf
Operating Officer ICOOI. This induded refining key data quality pro￿$$ documentation, and th$ d8velopmgnt ol a
comprehensive Key performan￿ Indicator IKPII dashboard, created by our Health IT partner.
The INPDR m8inlainecJ ils Is0￿01 accreditatson for Qu81ity Management Systems by the British Standards Instilutè
during this period lor all areas related lo dala collection and management This aCc￿ditation defflonstrgtes the INPDR
has robust System lor managing th8 registry and 5UPPOrting clinitsl research.
Changgs to INPDR 5t8ffing occurred during 2024125. Two new staff membèts join8d INPDR.. a full-tim8 Chi81
Operating offi.￿r ICOOI, who support5 cole r8gistry oFÈrations and activities, and 8 part-time Cornrnunicalions
Consultant who devek)ps and delwers INPDR'S communieatK)n approach to support the promotion of INPDR 8clivity and
progress to the p8tient Community and stakehothrs. The Chief Executive Officer ICEOI resigned snd INPDR as of
28th February 2025 In the CEO'S absence. the COO, with the support of the Board of Trustees, has been maintaining
business continuity. A repla￿ment CEO will be sought during 2026.
To 5UPPOrt achieving the aim of facilitating rèsearch, INPDR havè 8UPPOrt several applications for anonytnised
research data to facilitate resa3rch projects. These are 3ummarised below.
Research Studles
Sl Mary's Hospital M8nchester, UK, Oefining the seizure burden in Niemann Pick C."
- Aston University, UK. Quality ol life (QOLI in patients with Niernann-pick Disease Type C INPCI and thair car8rs.
University College London, Disease prog￿SsIon in early and late infantile Niem8nn-Pick.
University of Oxford. UK, The tnanifeslalions and m8nggement tsf inflammatory bowel d15ease in Niemann-Pid(
disease type C INPCI patients."
Univet5ily of Oxford, "Incorpoialion ofASIS into th8 INPDR lor NPC cases.
H05pilal for Sick child￿￿. Toronto, Canada, "Phenomenology ol movement disorders in N￿mann-PICk-O1Se&Se Type
C INPCI.. Findings from the Inlemational NietrTrann-Pick-Disease Registry IINPDRI
University ol Oxford, 3D-Modelling ol NPC1 mutations as 8 tool for disseetino disease mechanisms phenotypic
h8ter￿eneity
Pag8 5

INTERNATIONAL NIEMANN-PICK DISEASE
REGISTRY (REGISTERED NUMBER.. 1D6461291
Report ofthe Trustee8
forthe Year End&d 31 Ma￿h 2025
A¢hlev8ments and p8rformance
Gonfergnce Poster Presentations
Development of a novel palient-reported outcome IPROI measure for acid sphingorny81ina5e delKiency according to
the United States Food and Drug Adrninistralion IUSFDAI guidan￿ INORLDSymposium 2025, San Di8go, United
Slates ofAmerica
A Multi-Di5aplinary collaborats.on to Investigate Potential Contexts ol Using SleepBased DHTS in Clinical Research
Praclioe- WORLDSymposium 2025, San Diego, United St8te5 ofAmertca
Inve51igaling the Real-workj ExpeiEnce of Patients wth Niemann-Pick Disea88 Type C INPCI and Th￿r Carers
WORLDSymp05iurn 2025, San Diego, Unrted Statss ofAmerica
Reports
The impact ol treatment wrth olipudase alfa on adutt patients With ASMD - Part 2 Qualil8liv8 Report. Rare Disease
Research Partners.
Communlcatlon and engagement
INPDR ￿¢rUIted a Communication Consultant to SUPPOrt the promotion and visibility ol the work undertaken by th
chaiity Ihrough th8 development of a communication strategy and stakeholder engag8menl plan. This h85 alltywed
INPDR to have a greater presen￿ on sotlal media channels worldwide, alkymng communiCa￿onS to reach 8 ￿0bal
audien(
Rellecling the international scope of w￿, INPDR p￿sented re88ard) findirys arKI updates lo natK)nal and global
confe￿rn￿$, induding patient CCAnfflunty meetings..
- Niemann-Pick UK. UK
~Vaincre Les Maladies Lysosomales, France
- ASMO Espana, Segovia. Spain
- WORLD (We're Qrganising Research on LysosoThal Oiseas&l Syrnposium, Florid8, USA
Nalitsnal Niemann-Pick Disease Founclalion Conference, Florida. USA
- Ara Parseghian R8S6arch Conference, Arizona, USA
These Confe￿nceS not only provided an opportunity to showcase the work ol Ihe INPDR but also facilitsted direct
engagement with palignt community mernbets and stakeholders working in Niemann-Pick diseases. This engagement,
tum. increased INPDRS visibillty and •rfoTfn8nee in those Countr￿$.
Flnancial review
Flnancial posltlon
Unr8Stricled income in the year hwJs £371,210 (£2024 £39,143) and unrèstricted 8XP8ndrtute was £273,031 12024
£183.2491.
A restrict8d grant totalling £76,611 12024 £61,321) was award￿ by the Universlly ol Notre D￿8, Ihroygh ils Ara
Parseghian Medical Research Fund and Michael, Marcia, and Christa Parseghian Endowm6nt for Excellen{￿ in
Niemann Pid¢ Type C Research. The fund￿￿ received has en8bled the continuing develoynent of the Registry.
Alongside the high level ol support and Interaction from the patienl and clinical wmmunities, demon$lra18S the potential
of the Registry to make a posrtive d1ffefftn￿ for the global N￿mann-PIck patient community-
Reserves pollcy
There were free reserves of £11,387 on 31 March 202512024 -£7.4291 The board aim is to hold slx months running
costs in reserve which would be £136.515. Further as the r8glslry activit￿$ glow a huher lev81 of reser￿ will be
required.
Sustalnablllty & INPDR Gateway Ltd
A sustainability plan h8s been introduced with lh8 aim ol diversifying income and prov￿Ing financial stability. This
includes a commerci81 subscription m(Klel, a costs'ng model lor the rK)n-commercial s8Ctots plus a trust and corporate
gr8nt 5tiategy.
To support the commercial subscription, INPDR Gateway Ltrj was established. INPDR Gateway is a non-profit
commerci81 eompany established lo support and facilitate appropriate engagement between the Inlemational
Nlemann-Pick Disease Registry IINPDRI and industry partners. 51s primary purpose is lo provide a transparent,
strudured, and compliant mechanism through which Industry can engage with registry-related activities. while protecting
the independence, governance, and palIent-￿ntr&d mission ol the INPDR.
Page 6

INTERNATIONAL NIEMANN-PICK DISEASE
REGISTRY IREGISTERED NUMBER.. 106461291
Report of the Truste88
for the Year Ended 31 Mar¢h 2025
Flnancial review
INPDR Gateway enables industry partners to collaborate with Ihe registry on activities such as feasibilrty assessments,
data enquirie5. and research support. in a manner that is consistent ￿th ethical, leg81. and data-proteclKin
requir&ments By operating as a separate ￿gaI enlity. the Gateway allow8 commercial interaclK)ns lo ￿ msnag8d
appropriately, ensuring dear separation belweèn tharitaNe registry activit￿8 and commercigl58rvi￿$.
Importantly. INPDR Gateway ￿￿$ not compromise patient confidentiality or registry independence. All dats access is
governed by robust policies, wth oversight from the INPDR g0veman￿ structures, and no idenlifiabie patient data are
shared without aFvropriate approvals and Con$￿t.
Thiough this model, INPDR Gateway supports the long-lemi suslainalxlity of the registry, facilitates responsible industry
èngagement, and helps accElerate research and therapy devoloynent for N1eMann-P￿k dise88es, while maintaining
Iiust with patEnls, famili85. aThY IfÈ wider community. This financial year. INPOR Ga18way ctsntribuled £370.000
1£20.000 in 20241 to INPDR in reimbursernenl for setwces provided, maintaining the INPDR and en8bling further
development in line with priorits-es.
Future plan5
The INPDR has established a Solid foundation to achieve its airn5 and obje¢tivos through Ihe development of its
clinician-reported ancl pati8nt-reported databases which encompass88 ils registry system. ThrO￿ah data collected within
the iegiqtry systèm, high quality data can be 16ver8ged to enable impactful ¢linical ie58arch for Niemann-Pick diseases
for the benefit of p8ti8nls and families globally Our priorFty loi the coming year 15 10 5tr8nglhen this foundatK)n further to
increasa INPDR'S impa
For 2025126, 8 strategic anil op8ralional plan ha5 been defined, definlng 8 roadmap ol work that will further enhance th8
utility of INPDR'S registry data. Core work streams Include..
1. Research
INPDR will pu[sL￿ a number ol research opportunities. both intemally initiated 8nd in collaboration wth p8rtners, to
generate new knovAedge in Niemann-Pick diseases. Wthile the primary focu5 will rernain on levBiaging registry data,
INPDR will 8lso support research projects that initialty gen8rate data outside the iegistry system, with plans to integrate
those external datasets Into INPDR'8 repository.
2. Global Development ofCRD and PRD
Efforts will l(Kus on Incie85ing partEcipant recnjitrnent to both the Clinician Reported Database ICRDI and P8t18nt
Reported Database IPRDI. For the CRD, enh8nixd support rnea5uie5 for dini¢al sites will developecl, and updates
to the CRD IT system will be implemented to improve data utility. Materials will be produ{￿d lo help pab.ent community
organisatson5 expand CRD partiapation in *h8ir local territorie5, fvrtherenabling patient and Caregiver involvement.
Currentty, the PRD is available only in English. Work 15 scheduled to provide Ihe PRD in six langu8ges, enabli
broader global partiopalion. Cornplementino this. a PRD User Experience review will be ojnducled to identify
itnprovements that eTrhafi￿ dats utility and redu￿ barrErs lo partiapatiOTI.
3. Data Quality and Completènes
INPDR will strength8n registry data quality by improving its centr81 data monitoring process to identify and correct data
errors. A Key P8rforman￿ Indicator IKPII system will Le developed and launched lo allow for rea14imè monitoring and
view of CRD data. thereby enabling the tirnely intervention of data errors. ReguE8r data quality reports will be WodU￿￿
biannually to proV￿e insights into data integrity. ongoi￿ efforts will focu5 on improving data completeness anrl
aceur&y through refining data points. SOU￿ verification at clinical siles, and central data M￿ltOrI￿ @ffort.
4. Communication
Materials lo enable clear and nTreaningful communication outputs for INPDR'S diverse 518keholder audierte will be
produ￿d. Central to this will be the development of a 2025126 Communication Plan and Strategy, which will define
INPDR'S approach in engaging with its 5t3keholders. A schedule of r￿ular updates across all workstre8ms wll be
implemented, alongside targeled promotion of key outputs. INPDR will ￿entIfy and attend major conferences and
meetings lo disseminate findings within the global Niemann-Pid( communty and fosttrr cDllaboralions.
Structure, governance and management
Status and Govgmlng Document
INPDR is 8 company limited by guarantee goveiried by ils Memorandum and Atbcles ol Association. The company h85
one member, the Internationsl Ni8mann-Pid( Disease Alliance.
INPDR is èlso ￿91s(ele{l a5 a charity with tha Chaiity Cornmission.
Page 7

INTERNATIONAL NIEMANN.PICK DISEASE
REGISTRY IREGISTERED NUMgER.. 106461291
Report of the Trustses
for the Yèar Ended 31 March 2025
struclure, governance and management
Recruitment and appolntmenl ol n8w trustees
Future Tiustees shall be appointed by the TIu5tees from lime to time lolk)wing a nomination received from th8
Nominations Committee. TIust8es are volunt88rs and each takes on responsibilrtie5 within the organisation to
co-ordinatelsupport an aspect of its function.
Inductlon and trdinirEg of newtrustees
New Trustees are briefed on their legal obligstions, Ihe wntenls ol the memorandum & artides of 8ssocialion, th8 board
and decision tnaking processes, the business ￿an and the iecent finanaal p8rfOrMan￿ ol the charity. During the
inductic￿. Ihey are mentored by other Trustees.
Oragnlsational Stru¢tur
The Trustees ar8 d1￿CHY responsiblè for the effertiV8 govemance of th8 organisalion in accordan￿ wlh stalutory
guidance and legislation as provided by the Charity Commissioners and Companies House. The Trust8es tneet
quarterly wrth additional contact as required, in order to r8view reports and to carry out management and financi81
reviews.
Tiustees follow a code of conduct, which promdes dear guidelines as to the standards ol behaviour, responsibilit￿,
and best practice expected of tho58 involved with Ihe INPDR.
The Trustees del￿at@ th8 day-to-day management of th& oiganisation to lh8 Registry Manag8m8nt Team, by the
Chief Executive Offi￿r. who reports regularty to thtr 8oard al quarteily virtual meetings.
Key management remuneratlon
All trustees give of their time freely no trustee receiverl remun8ration in the year. The board is responsib￿ for
Setting ￿Trployee s8lary ￿Ve1$. All sal8ri8S S￿ benchmarked wlh smilar roEe5 in the voluntary secior, not with th8 public
or private sectors The size ol U)e organi5ation Is also Consid81￿.
Wider nelwork
The chgrity's tnember is the Int8fflatv)nal Niemarin-Pi¢k Dise83e Alliance IINPDAI which is an alliance of non-profit
5UPPOrt organisations who are asso￿ated with the iare group of g8n81ic diseases known collectively as Nietn8nn-Pick
Disèases. The Alliance provides a foium where. through the exch8tvJè of infomiatK>n. experien￿ and kn￿￿ledge,
global progress caD be aecelemted
Page 8

INTERNATIONAL NIEMANN.PICK DISEASE
REGISTRY (REGISTERED NUMBER.. 106461291
Reptsrt ofthe Trustees
for the Year Ended 31 March 2025
Structure, governanc& and management
Risk managom&nt
As part of the Quality M8nagement Systern. which has IS09001 accr8dilalion, the INPDR rnaintains a risks and k8sues
109. Risks are tnanaged on an ongoing ba818 by the Registry Management Team al￿ reviewed by Boarrl Members at a
qU8rterly Management RevEw Meeting.
The reglstry operates in 8 highly iegulated din￿al research environment a￿1 must ansur8 Cornplian￿ with data
prolethon law and good dinical practi￿ INPDR operates in Complian(￿ with the European M&Sicine Agency IEbAAI
Guidelines on the Managem8nl of p￿t￿nI Registli88 and Ihe regi51ry has received a ￿tter of support Iiom EMA as a
res(xJrce for dinical ￿searCh.
To manage r￿kS relat8d to data protection and ensure the privacy of registry participants, INPDR employs the servic6S
of a consultant Data Protection offi(￿r. In addition, during the year, our teehnology partnei, Op8nApp was awarded
IS027001 aecr8ditation for dats sscurty, providing further Confiden￿ th8t the INPDR are wellylac8d lo ensu￿ patient
priv£ey.
th r8g8rd to data protedion. there were tK) atherse even15, incKlents or breaches r8POrted during 2024125 no
data su4.ect rights requests.
Principal Rl$ks and Mitigatlon . INPDR
The prinap81 rigks ants'¢ipated in the foreseeable future, logether with mitigating actions, include."
Suslalnabillty and fundlng rlsk
Risk." Limited and uncertain lon*temi fundiw may affect the ongoing operal'on, maintenan￿. (levelopment of the
regislry
Mih"g8tion.' INPDR actively seeks diversifi8d funding sourcss. including grants. Partnerships, and collaborative prqects,
and priorilises act1Vlties that maintain coi8 iegistry functionality and data quality.
Patient enrolment and geographlc iepresentatlon
Risk.. Insufticienl patient enroltn@nt or uneven getsJraphi¢ repRsentstK)n may limit the robu5tn&85. generalisability, and
utility of registry data.
Mitrgalion.. INPDR woik5 d0881y with Pat￿￿1 organisations, diniuans. and advocacy ne￿orkS (including INPDA
memb8rsl to encourage enrolrnent, raise awsren8ss of the registry, and support part￿pation acmss diverse 18gK)ns.
Data quality, completen955. and conslstency
Risk.. Variability in d8t8 entry, incomplet8 datasets, or inconsistent reFX)rting may redu￿ the scientiric and clin￿al valua
of registry outputs.
Miligalion.. INPDR applies defined d8ta standards, prowd&8 gUVsan￿ and training lo participakn'rYJ sites, and undertakes
iegular data quality monrtoring and validation.
- Data protection, privacy, and cybersecurlty
Risk.. Brea¢h ol dats protection regulations or cybersecurity IneK18nts o)uld compmmi5e sensitive patlenl data and
damag8 trust.
Milig81ion.' INPDR operates in compliance wth relevant dsta protectk)n regulations linc1￿ing GDPRI, etnploys robust
d8ta security measur8S, 2nd regularly ievi8w$ governance, consent pro￿se$, and lechnul safe-uards.
Dependence on cllnician and site engagement
Risk". ReliaTTrce on dinician participation and site 8ng8gement may affect data subrnis510n rates and I0￿-terM
suslainakn-lity-
Mitigation.. INPDR mailltai￿ 8Ctive engagemènt with dinical sites, demonstrates the value of registry participation
through outputs and f*dbad<. and seeks to reduce adminislrah.ve burden where p055ibkg.
Regulatory and ethlcal ¢omplex¥ty
Risk." Vaiiability in natlOn81 iegulalory and ethical requirements may slow site onboarding or limit participation in certain
jurisdictions.
Mitigation.. INPDR piovid8S SUPPOrt to sites navigsling loc81 approvals and adapts processe5 wh8re feasible io
accommodate differing r8gvlalory en%4ronments
Governance and operational ¢apa¢lt y
Risk.. Limited operational capauty m8y constrain Fegistry development, 18spon5iveness io t8searo* requests, or
expansion o15cope
Mitigation.. INPDR maintains dear g0Veman￿ structures, indudiru Board and Scientific Advisory Committee ov8rsight.
and prloritises activities aligned with slr8legic objective8 available resources.
Addltlonal Risks Con81dered
Stskoholder allgnment and expectatlons
Risk.. Differing expectations amorvJ patients, clinicians, rès6arth8rs. and industry Partners may e￿ate thalleryes in
prioritisalion afid data use.
MitvJalion.' INPDR applies transparent governan￿ processes, cP8ar data acc888 wli(#es, and Inclusive stakehokjer
engagement lo align 8xpeclalions.
Pag8 9

INTERNATIONAL NIEMANN-PICK DISEASE
REGISTRY IREGISTEREO NUMBER.. 106461291
Report ol the Trustees
for the Yèar Endad 31 March 2025
structure, goveman¢e and rnanagement
Reputational risk
Risk.. A5 a trusted sourcè of disease-speafic data. INPDR fa￿S reputatK)nal risk related lo data use, partnership5, Dr
pe￿IVed conflicts ol inleie5t.
Mitigation.. INPDR maintains independen￿, a￿lieS rigorou5 ￿VIeW of data aC￿sS iequ85ts, and ensures transparency
in partnershps and comrnunications.
Rectsgnising the contrlbutlon of volunleers
We ale extremely grateful tts those who give their tin8 as volunteers in support of the INPDR. Wfithout their dedicated
and conlinu8rS support, w8 would not be able to achieve all that is required on Lehall ol our orgAnisation and community
Tris report has been prepared in aecordance with speci81 provisions of Part 15 of the C(mpanies Act 2006 ￿lating
lo srnall cornpanies.
Approved by order ofthè board of trustEes on 19 D8C8rnLEr 2025 and Signed on its b8h81fby'.
J E Gre8n - Truste8
Page 10

Independent Examlnerfs R8POrt lo the Trustees of
International Nlemann-Pick Dlsease
Reglstry
Independent examln&Vs report lo the trustee5 of International NiemanTt-Plck Dlsgase R8glstry I'the Company'l
l ￿pOrt to the charity trustees on my examination ol the accounts of the Company for Ihe year ended 31 March 2025.
Responsibilities and ba515 of report
As the chgrity's tiustees ol the Comp8ny land also its directors for the purposes of company lawl you a￿ responsible lor
the preparation of the a¢¢ounts in 8ccordance with th8 requirements of the Gompanies Act 2CO61'the 20Qb Act'i.
Having satisfied myself that t1￿ 8ccounts of the Company aitr not required to be audited U￿er Part 16 of the 2CQ6 Act
and are eligible for independent examination, I report In respect of my examination of your charity's accounts as cairied
out utKJer SectDn 145 of the Charities Act 2011 I'the 2011 Acl'l In ¢arryitvJ out my examinatK)n I have fdbwed the
D￿eCtionS given by th& Charity Commission under sects.on 145151 Ibl ol the 2011 Act.
Independent examinerfs statement
sin￿ your charity's gross incorne ex￿eded £250.000 your exatnin8r must be a [nErn￿r of a listed body. I can confirm
that l am qualified to undertake the examination because l am a member of the Insb'tute ol Charteied Acwuntants of
Scolland, whi¢h rs ofthe listed bodies.
I have completed my examination. I confirm that no matters have corne to my attention in ￿nnectIon with Ihe
exarrinalion giving rne cause to be[E￿..
accounlirTrg r9cords wer8 not kept in resped of the Company a5 required by Section 38e olthe 21K)6 Act,. or
the ac¢ounts do not 8ccord with those records., or
the accounts do nol comply with th8 awounting fftquirernents ol Section 396 01 the 20ffi Act olher than sny
requirement that the accoun15 give a twe and fair view which is not a matter considered as part of
independent examination,. or
the sccounts have not been Prepa￿d in accordance with th8 rnethods and principles ol the St&t8ment ol
Recommended Practice for 8ccounting and ￿pOrting by charities (applicab￿ lo charities prep8ring their
ccounls in accordan￿ with the Finan￿￿1 Reporting Standsrd 8pplical￿e In the UK and Republic of Ir8land IFRS
10211.
I hav8 no cOn￿rnS and have come across no other matter5 in connection with the examination to which attention Sh￿k1
be drawn in this report in order to enabl8 a proper understanding of the aeeounts to be reached.
Christopher T Spalding
The Institute of Chartered A¢count8nts ol Scotland
Jatnes Anderson & Co
Charteied A¢counlants
Pen118nd Eslale
strailon
Edinburgh
EH20 9QH
19 De￿mber 2025
Page11

IprrERNATIONAL NIEMANN-PICK DISEASE
REGISTRY
St4tement of Finanelal Activititrs
Ilncorporating Income and Exptrndlture Accounti
for the Year Ended 31 Mar¢h 2025
2025
Total
funds
2024
Total
funds
Unrestrlcted
rund
Restrlcted
fund5
Note$
Incomo and endowmonts from
Donations and legacies
78,611
76,611
.875
Charitable aetlvitles
Chaiitable activities
371,210
371.210
38,623
Total
371.210
76.611
447,821
131.498
Expendlturb on
Charltable activities
Ch8ritÉble aciivi118S
273,031
95,427
366,458
307,686
NET INCOMEIIEXPENDITUREI
98,179
116,8161
79.363
1176,1881
Reeonclllatlon of funds
Total funds brought fopNard
17,4291
18,816
11,387
187,575
Total funds carrled fotward
.750
,750
11.387
The note5 form part of these financial statements
Page 12

INTERNATIONAL NIEMANN-PICK OISEASE
REGISTRY (REGISTERED NUMBER.. 106461291
Balance She8t
31 March 2025
2025
2024
Notes
Flx&d as8et8
Investments
Current assets
Debtors
Cash at bank
10
56,111
68,007
22
25,363
124.118
25,385
Crèditors
Amoun15 falling due within one year
133,3691
113,9991
Net currant as8ets
,749
11,386
Total a$$ets less current Ilabllltlgs
90,750
11,387
NET ASSETS
90,750
11,387
Funds
Unrestri¢leLI funds
Restricted funds
13
90,750
17.42gi
16.81e
Total funds
,750
11,387
Th8 charitable company is enlitled to exemption from a￿11 undèr S8cbon 477 01 tr￿ Companies Act 2W6 lor the year
ended 31 Maich 2025
The metnbers have not wuired company to obtaln an audit ol its fin8n(ial statements for the year ended
31 Maich 2025 in a￿ordance with Section 476 01 the Companies Act 2LK6.
The trustees acknowledge their responsibilities for
ensuring that the charitable company keeps accountirrfJ records that cornpty with Secb'ons 388 and 387 of the
Companies Act 2006 and
preparing financial slalemenls which give a true and fair view of the state ol affairs of the charitable cofflpany as
al the end of each finanaal year and ol its surplus 01 defi¢it for each finall￿al year in accordance wlh the
reqLJirernenls ol Sections 394 and 395 and which oth@rwise Comply with the requirernents ol the Cornpanies Act
2006 relating lo financial statements, so lar as applicab￿ lo the charitable cornpany.
Thé89 financial statements have been prepared in accordan￿ with the wovisions applicablè to tharitable companies
subject to the small comp8nies regime.
Ibl
The finanual st8temenls were approv8d by the Board of Trustees afKI aulhorised lor issue on 19 December 2025 and
were sKJnEd on its behalf by..
J E G18en- Trustee
The notes fom part of these finar￿la1 5taternents
Page13

INTERNATIONAL NIEMANN-PICK DISEASE
REGISTRY
Notes to the Flnancial Statements
for the Year Ended 31 March 2025
Accountlng pollcles
Basls of pr8parlng the flnanclal ststements
The finanual statements of th8 charitable cornpany, which is a public benefit entity under FRS 102, have been
prepar8d in awoidance with the Charities SORP IFRS 1021 'Accountin9 and Reporting by Charilies". Slatemenl
ol Recommended Practits applicab￿ to d)arilie5 preparing t￿1r accounts in accordan￿ with the Financial
Reporting Standard applicable in UK and Republic of Ireland IFRS 1021 leffectiV8 1 January 20191,, Financial
Reporting Standard 102 'Th8 Financial Reporting Standard applicable in the UK and Republic ol Ireland, and th8
Companies Act 2006. The finanaal statements have been prepared undei the hisloiical cost Convention. wrth the
ext￿ptIOn ol investments whKh are induded al market value.
Inlemational NIeman￿Pick Disease Registry meets the definition of a public benerrt entity undgr FRS 102.
In¢omè
All income is recognised oncè tt)e charity has entitlement to the incorne, there is sufficient ￿rtaInty of ￿￿1pt
and SD 11 is probab￿ that the income will be received, and thè amount of income receivable can be me85ured
reliabty.
Donation5 and grants are reeognised whell they have been communlcated in writinu with notification of both th8
arnount and seluemenl dale. In the ev8nt that a donatK)n or oranl subject to conditions that require a level of
p&rforman￿ b8fore the charity 15 entitled to the funds. the income is doferred and not recognised until either
those conditions are fijlly met, or the fulfjlmenl of those condilions is wtK)Ily within the ¢ontrd of the chaiity ar￿ rt
is probab￿ that tho58 conditions will be fulfilled in the reporting period.
Expenditure
Expenditure is re￿g￿lS8d as soon as t￿re is a legal or constructive obliqation (x)mmith'ng the eharity lo thal
8xpendilure, it 15 probable that a trnnsfer of economio b8nefits will b8 18quired in settlement and the amount of
Ihe ￿ligatIon can be measured rdiably.
All 8￿r￿ltUre is accounted for on an a¢crua15 basis. All expenses, including support costs and goveman
costs. are allocated or apportioned lo the applicable expenditure headings in the Statement ol financial activities.
ExpenditurÉ on charitable activities inrjudes any VAT which cannot be ￿cOvered and is reported as part ol th8
8xpendilur8 to which rt relates and Compiises those costs incurred by the charity in the delivery of its actiwlies
and servi￿$ for its benefi￿a￿&s. 11 includes both Costs that can be allocated directly to such a¢liviti88 atKJ those
costs ol an iThlirect nature necessary to support them.
Taxation
The thaiity Is exempl frotn corporalkjn tax on its charitable activities.
Fund accountlng
Unreslricled funds can be used in ac¢ordan(x with the charitable objectives atthe discretion of the truste9S.
Restrieted funds can only be us8d for particular restricted purposes wthin the objects of th8 charty. Resttithons
arise wlKn specified by (k)noror when funds are raisgj lor p8rticular reslrir18d purposes.
Further ex￿anatiOn of the nature and purpose oleach fund Is included in the notes to the financial ststements.
Pension costs
The wmpany operates a defined contribution scheme, the 8ssets ol which are held s&patately from those ol the
¢harity.The pension eost d)arg8 tepresenl$ contributions p8yabk by the chanty to the scheme.
Investments
The investfflent in the group undertaking 1$ measured at cost less Impairm&nt on the basis trat it repre58nts
shares in all enl'ty that is not publKly tr8ded and the falr value cannot otherwise b8 measured
re11abty.
Debtors and credltots r8celvable I payable within one year
Debtors and creditors wlh no stated interest rate and ieceivable or payable within one year are r￿Orded al
transaction price Any losses arising from impaimienl are r8cognised in 8xpenditure.
Cash In bank and In hand
Cash at bank and cash in hand includes cash and short term highly liquid investments.
Pag8 14
continued.

INTERNATIONAL NIEMANN-PICK DISEASE
REGISTRY
Notes to the Flnanclal Statèments - continued
forthe Year Ended 31 March 2025
Accounting pollciès - contlnued
Goln9 ¢onterA
The finall￿81 slalements have been prepared on a going con￿rn bask 85 the trustees believe that no material
uncertainties exisL Th8 Iruste&s have considered the level ol fund5 h8ld and the expected ￿vd ol incorne and
expenditure for 12 months from aulhorising these financ4al statements. The budgeted income and expenditure is
sufficient with the level of reserv8s for the charty to be able to contlnue 8$ a going concern.
Donatlons and legacies
2025
Total
fut)ds
2024
Total
funds
Unrestrlcted
funds
Restrlctpd
futTrds
Donalk)ns
Grants
520
92,355
78,611
76,611
76,611
76,611
92,875
Grants re￿ived. included in the above, are as ft)Ilows.'
202$
2024
Sanoli G8nzyme
Universty of Notre Dame Lac
National Lottery Cornmunity Fund
11,534
61,321
19,500
76,611
76.611
92,355
Income from charitable actlvities
2025
2024
A¢tlvity
Charitable activrtSes
Contribution to Re58a￿h
Recharge lo INPDR Gateway
Ltd
Third party reimbursement
18,459
Charitable aclivib'es
Charitable activities
370,000
1,210
20,OCX)
164
371,210
38.623
Charitable actlvllles costs
Dlrect
Costs (see
note Sl
char￿￿bI8 activities
368,458
Pag& 15
conb'nued...

INTERNATIONAL NIEMANN-PICK DISEASE
REGISTRY
Note5 to the Flnancial Statements - contlnued
for the Year Ended 31 March 2025
Dlrecl costs of charltabl8 aetivltles
2025
2024
St8ff costs
Sundrifjs
Equipment costs
Registry hosting
Travel & meetings
Website costs
Olher staff costs
Office 8xpen5es
Promotional expenses
Executive meetings
Iridependent examin6¢s fee
Other reguiqtory ¢05ts
Consultancy
Research
HIC m91nt8nan￿ & supFM)rt
Patient Facing Acts-vities
INPDR Gat￿waY
171,022
129,563
1,860
48,663
21,349
1.295
1.947
9.011
21,740
29,722
1,027
9,520
5,826
2,424
1,974
3,713
71.669
20,068
10,440
3,413
1,512
75,840
10,749
11.520
5,643
23
368.458
307,686
TTU5tègs' remuneration and benefi18
There were no tru5tees' remuneration or other benefits for the period ended 31 March 202512024- nil)
Trustees. expenses
Duriro the period one trustee was reimburs8d £14012024 £5831 lor travel exp8nses.12024- 11
Staff costs
202$
2024
Wages and salaries
Soual security costs
other pension costs
159.320
9,022
121,223
5,993
2,347
171,022
129,563
The average monthty numberolemployèès during the yearwas as follows..
2025
2024
CcM)rdin8b"on and adminislr8tion
Registry M8n4er
The Chief Execubve Offi￿r is key management and his total employee tEnefits (including Nl and Pension) were
£60,465.12024 £65.4551
Page 16
continued...

INTERNATIONAL NIEMANN-PICK DISEASE
REGISTRY
Notes to the Flnancial Statements. Contlnued
for the Year Ended 31 March 2025
Comparatives for the statement of flnancial actlvltles
Unrestrlct•d
fund
Restrlcted
funds
Total
funds
Incomè and gndowments from
Donations and legacEs
520
92,355
92,875
Charitable actlvltles
Charitable adivilies
38,623
38.623
Total
39,143
92,355
131.498
Expenditure on
Charltable actlvltles
Charitable actimlies
183,249
124,437
307,686
NET INCOMEIIEXPENDITUREI
1144,1061
132,0821
1176.1881
Reconciliation of fund5
Total funds brought forward
136,677
50,898
187,575
Total funds carried forward
17,42gi
18,816
11,387
Fixed asset Inv￿lM￿nts
Shgras In
group
undertakings
Market value
At 1 April 2024 arKJ 31 March 2025
Net book value
At 31 March 2025
At 31 March 2024
Th8r8 were no investmènt assets outside the UK.
The companvs inv851ments at the balanc8 sheet dale in the shale capital ol ccthpènie5 include the following..
INPDR Gateway Ltd
Registe￿￿ offi￿. United Kingdom 1270631
Nature of business.. Data WOVKler for research
Class of Share..
holdlng
loo
2026
2024
Aggregate capital and r858rves
Profitlllossl for Ihe ye8r
142.2471
6.563
149.4461
152.1601
P8ge 17
continued...

INTERNATIONAL NIEMANN-PICK DISEASE
REGISTRY
Notes to the Flnancl81 Statements- contlnued
for the Y•ar Ended 31 March 2025
10.
Debtors- amounts falling due withln one year
2025
2024
Trade debtors
Prepayments
55,(MX)
22
56.111
22
11.
c￿dItorS. amounts fallln9 due within one year
2025
2024
Sccial Security and other taxes
Accruals and deferred incThne
2.421
30,948
2.854
11,145
33,369
13.999
12.
Analysis tsf neta$$ets between funds
2025
Total
funds
2024
Total
funds
Unrestrleted
fund
Re8tricled
funds
Investments
Current assets
124,118
133.3691
124,118
133,3691
25,385
113.9991
90,750
90,750
11,387
13.
Movement In funds
Net
mov8m•nt
In fund5
At
3113125
At 114124
Unre$trlcted funds
Geneffdl fvnd
17,4291
98.179
gJ,750
Reslrtcted funds
APMRF
National Lottery Community Fund
$34
18,282
15341
118,2821
18,816
118,8161
TOTAL FUNDS
11,387
79,363
90,750
Net movemgnl in funds. induded in thè atrrt)ve are as follow5.'
Incoming
rgsources
Resources
expended
Movement
in lund5
Unrestrfcted funds
General fund
371,210
1273,0311
98,179
Re5trleted funds
APMRF
National Lottery Communily Fund
76.611
177.1451
118,2821
15341
118,2821
76,611
195,4271
118.8161
TOTAL FUNDS
447.821
1368,4581
79.363
Page 18
continued...

INTERNATIONAL NIEMANN.PICK DISEASE
REGISTRY
NDte5 to the Flnanclal Statfrments- contlnued
for the Year Ended 31 March 2025
13.
Movement In funds - Contlnued
Comparatlves for mov8mont In funds
Net
mov$m8nt
in fund8
At
3113124
A¢ 114123
Unrestricted funds
Genera1 fund
136,677
1144,1061
17.4291
Restricted funds
APMRF
Nation21 Lottery Cotnrnunity Fund
50,898
150,3641
18,282
534
18,282
50,898
132,0821
18,816
TOTAL FUNDS
187,575
1176.1881
11.387
Comparative nel movemènt in furKls, included in thè above are as follows..
Incomlng
resources
Re$ources
expended
Movement
In fund8
Unre$trlcttd funds
G8neial fur
39.143
1183,2491
1144,1061
Restrlcted fund
APMRF
Sanofi
National Lottery Community FurKI
61.321
11,534
19,500
1111,6851
111.5341
11,2181
I￿.364)
18,282
92,355
1124,4371
132,0821
TOTAL FUNDS
131.498
1307,6861
1178,1881
The general fund 15 fre8 to use in accordan[￿ with the objects olth6 Charity.
The APMRF fund is to aC￿lerate progr6s5 by fa¢ilitatiw regFS1ry recruilmenl in the USA.
Th8 S8nofi fund was towards the INPDR 2023 Forum
National Lottery Cotnmunily Fund is for telli￿ the Story ol Niemann-Pi(Jt Diseases in UK
14.
Related paity dl$¢losures
The Inteinalional Niemann-Pick Oisease Allian￿ IINPDAI is the only member ol the cotnpany During the year
costs were ch8rged by INPDA to INPDR of £Nil 12024 £3,014). INPDA p8id shared costs of £4.991 12024
£4,422). Al 31 March 2025 there wa5 a balan￿ due to INPDA of £Nil 12024 £Nill.
Niemann-Pick UK INPUKI Is a member of the INPDR. During the year there weie r8charges lor shared Staff.
travel and overtE3ds etc by NPUK to INPDR of £40,047 12024 £27,142). INPDR ￿e￿ived income in the year
from NPUK ol £NIl 12024 - £7.9811. At 31 Msrch 2025 there was a balance du8 to NPUK of £3,560 12024
£2,650).
The INPDR (R￿15tryI fully owns a C￿MpaNy, INPDR G3teway Ltd (Gateway), whith carries out the commercial
opeialions of the R￿15try. Djring the year 8 sum of £Nil12024 £Nill was lent by the Registry lo the Galewsy. In
addition recharges and Servi￿ charge5 were made lo the Gateway iii the year of £370,(K)0 12024 £20.7611. A
sum of £55,[￿0 was due from the Registry at 31 Mar¢h 202512024 du8 to £221.
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