## **Trustees’ Annual Report for the period** ## **From 01/04/2024 To 31/03/2025** ## **Charity name: Cure DHDDS** ## **Charity registration number: 1202643** ## **Objectives and Activities** ||SORP reference|| |---|---|---| |Summary of the purposes of
the charity as set out in its
governing document|Para 1.17|The objects of the CIO are to relieve the needs
of those affected by DHDDS Disease to support
families affected by DHDDS mutations and
drive research into them| |Summary of the main
activities in relation to those
purposes for the public
benefit, in particular, the
activities, projects or
services identified in the
accounts.|Para 1.17 and
1.19|**Families Support/Information Services**
The charity provides a support group for
individuals and families affected by the
condition. We aim to provide the latest
information on the condition to families and
health professionals via our website, e-news
and social media.
**Educational Activities**
We continue to work on raising both public
awareness and education amongst key
medical professionals throughout the world
so they can relate to the signs, symptoms
and medical diagnosis of a DHDDS gene
mutation.
Speaking at and/or hosting exhibition
stands (in-person or virtually) at medical
and scientific conferences provides
opportunities to fulfil our aims and
objectives whilst developing strategic
partnerships with research facilities,
medical and pharmaceutical institutions,
and like- minded charitable bodies.
**Research**
The charity has raised funds to pay for
research to be undertaken by Sainte Justine
Hospital, UCL Cardiff University and the
ICAHN School of Medicine. There are a new
research projects that we intend funding in
2025/26.| Statement confirming Para 1.18 The Trustees consider the charity’s activities whether the trustees have are wholly in the public interest and had regard to the guidance designed to benefit all those who suffer from issued by the Charity DHDDS, their families and medical Commission on public benefit professionals dealing with this debilitating condition, in line with the Charity’s Commission guidance on ‘public benefit’. ## **Achievements and Performance** SORP reference Funding/Fundraising Summary of the main Para 1.20 achievements of the charity, identifying the difference the charity’s work has made to the circumstances of its beneficiaries and any wider benefits to society as a whole. We have once again been overwhelmed by the generosity that we have received from donors this year. Notable fundraising events included the John O’Groats to Lands’ End Tandem Bike ride, the Home Guard DHDDS Summer Party, London Scottish Auction, the Midtown Business Club’s Golf Day, Sophie Clegg’s 3 Peak Challenge, Katie Barr’s Brecon Beacon trek plus several independent fundraisers. **Research** Sainte Justine Hospital, Montreal – We supported a second year of Mouse model research with Alexey Pchejetski. University College London (UCL) – We funded preliminary research into an RNA therapy and subsequently Dr Raj Karda and Dr Ozge Cetin have applied for a grant to undertake AI-Based Preclinical Studies Utilizing AAV9-Mediated DHDDS-Specific Gene Therapy for Ultra-Rare DHHDS Disease. ICAHN School of Medicine – We have supported the development of IPSCS and DHDDS brain organoids for disease analysis and screening of NAD precursor treatments. This |has been translated into an international| |---| |clinical trial under the supervision of Prof Eva| |Morava| |Cardiff University - Prof. Emyr Lloyd Evans and| |Dr Hannah Best – we have funded a 19 month| |research program focused on repurposing| |small molecule drugs developed for Niemann| |pick c disease in DHDDS patient cell models| |We continue to work very closely with Cure| |DHDDS USA on current and future research| |projects in the US including the development of| |Brain Organoids at Mount Sinai Hospital, the| |development of ASOs at n- Lorem and| |developing NAD precursors as potential| |therapies for DHDDS and NUS1 patients| |Conferences/Events attended + speaker| |opportunities:| |We hosted another two incredibly successful| |Cure DHDDS conferences in conjunction with| |Costello Medical. The very first conference was| |the winner of The Communique Awards 2024.| |There were 53 attendees at the Roundtable in| |April 2025 and then 70 attendees are expected| |at our Cure DHDDS Scientific Exchange Meeting| |in October 2025| |Mel has actively represented Cure DHDDS at| |numerous events in the last financial year both| |online and in person — to raise awareness of| |our mission and accelerate research. She has| |been featured in a range of podcasts, videos,| |and conferences, including:| |
Behind the Genes Podcast – Genomics| |England| |
UNIAMO Podcast – Focus on DHDDS| |
Rare Disease Research Network| |
Patient Centricity Congress – One| |Nucleus| |
Festival of Genomics| |
Orphan Drugs Congress Europe 2024| |
7th Patient Centricity Congress 2025| |
UPNAT Symposium| |
Genomics England Research Summit| |
Aspire Biosciences| |
N-Lorem Colloquium| |
Therapeutic Innovation and Trials –| |UCL| |
UK Platform for Nucleic Acid Therapies| |(UPNAT) Inaugural Symposium| |
Oxford Harrington Symposium| In addition to these events, Cure DHDDS has received media coverage from major outlets, including ITV London, _The Independent_ , _Daily Mail_ , _The Sun_ , _Citywire_ , and others — helping to significantly raise public awareness and accelerate research progress. Cure DHDDS has set up a patient registry with Cords at Sanford Cure DHDDS benefits from services provided by Costello Medical including mentoring, training, and logistical support We remain committed to supporting our rare disease community, many of whom have felt deeply isolated and underserved. Our private Facebook group now includes over 170 affected families, providing a safe and supportive space for connection and shared experiences. We have hosted several online meetings, bringing together both our international patient community and leading researchers to foster dialogue and collaboration. In addition, we regularly share research updates in both written and video formats, ensuring families stay informed and engaged. ## **Financial Review** |Review of the
charity’s
financial
position at the
end of the
period|Para
1.21|Thanks to ongoing support from individuals and companies, the
charity is in a strong position at the end of its second financial year.
For the 12 months to the year end (31stMarch 2025) the charity had
receipts of £131,495. Cash funds at the end of the financial year were
£221,262.20.
We have made four grants this financial year, with significant future
commitments to Cardiff, Sainte Justine, UCL and Birmingham
University as detailed below:
£0.00
£5,000.00
£10,000.00
£15,000.00
£20,000.00
£25,000.00
Grants and Costs 2024/25| |---|---|---| |Statement
explaining the
policy for
holding
reserves
stating why
they are held|Para
1.22|The purpose of the reserves is:
 To fund working capital and time lags in the receipt of
promised funds
 To fund unexpected expenditure, e.g., when projects
overrun, or unplanned events occur
 Manage economic or regulatory changes which may
have unforeseen financial consequences
We assess the level of general reserve needed by looking forward
and considering the risks to our funding balanced against our
expenditure commitments. To ensure we can continue to operate
in accordance with our plans, we would look to hold a general
reserve in the range £10,000 - £12,000 to cover unfunded
committed costs for the next 6 months.| |||| |---|---|---| |Amount of
reserves held|Para
1.22|Our reserves as of 31 March 2025 were £221,662| |Reasons for
holding zero
reserves|Para
1.22|| |Details of fund
materially in
deficit|Para
1.24|| |Explanation of
any
uncertainties
about the
charity
continuing as a
going concern|Para
1.23|| **Additional information (optional)** You may choose to include further statements where relevant about: The charity’s principal sources Para 1.47 of funds (including any fundraising) ## **Structure, Governance and Management** |Description of charity’s
trusts:||| |---|---|---| |Type of governing document
(trust deed, royal charter)|
Para 1.25|CIO association constitution| |How is the charity
constituted?
(e.g unincorporated
association, CIO)|Para 1.25|CIO association constitution (formed 06 April
2023)| |Trustee selection methods
including details of any
constitutional provisions e.g.
election to post or name of
any person or body entitled
to appoint one or more
trustees|Para 1.25|By appointment of registered members at
general meetings and by Trustees| |**Additional information (optional)**
You may choose to include further statements|**Additional information (optional)**
You may choose to include further statements|where relevant about:| |---|---|---| |Policies and procedures
adopted for the induction
and training of trustees|Para 1.51|New trustees are asked to abide by a
Code of Conduct and are issued with
Essential Information for Trustees from
the Charities Commission, sign a Trustee
Declaration and complete a Fit and
Proper persons form.| |The charity’s organisational
structure and any wider
network with which the
charity works|Para 1.51|Cure DHDDS is run on a voluntary basis.
Trustees consult on all significant matters
of policy formulation, family support and
methods of communication about Cure
DHDDS information services to medical
professionals.
The Scientific Advisory Board remains in
place with 15 leading scientific advisors
helping us understand the research and
determine the most relevant use of
funding.
The charity operates the following
policies: child protection, data privacy, risk
management and volunteer management.| |Relationship with any
related parties|Para 1.51|**The charity is a member of:**
Cure DHDDS and NUS 1
Community
Genetic Alliance UK (GAUK)
Rare X
UK Rare Epilepsies Together
network (UKRET)
CDG Care
CDG Global Alliance
CDG UK
LSD Collaborative
• The Charity works closely with Cure
DHDDS USA| Other ## **Reference and Administrative details** |Charity name|Cure DHDDS| |---|---| |Other name the charity uses|| |Registered charitynumber|**1202643**| |Charity’s principal address|6 Park Avenue
London
SW14 8AT| ||| ## **Names of the charity trustees who manage the charity** |1
2
3
4
5
6
7
8
9
10
11
12
13
14
15
16
17
18
19
20|**Trustee name**|**Office (if any)**|**Dates acted if not for whole**
**year**|**Name of person (or body) entitled**
**to appoint trustee(if any)**| |---|---|---|---|---| ||Melanie Dixon|||| ||Charles Dixon|||| ||Victoria Allan|||| ||PennyBrogan|||| ||Louise Townley|||| ||Katie McCombe|||| |||||| |||||| |||||| |||||| |||||| |||||| |||||| |||||| |||||| |||||| |||||| |||||| |||||| |||||| ## – Corporate trustees names of the directors at the date the report was approved **Director name** Name of trustees holding title to property belonging to the charity **Trustee name Dates acted if not for whole year** ## **Funds held as custodian trustees on behalf of others** Description of the assets held in this capacity Name and objects of the charity on whose behalf the assets are held and how this falls within the custodian charity’s objects Details of arrangements for safe custody and segregation of such assets from the charity’s own assets ## **Additional information (optional)** ## **Names and addresses of advisers (Optional information)** |**Type of**|**Name**|**Address**| |---|---|---| |**adviser**||| **Name of chief executive or names of senior staff members (Optional information)** ## **Exemptions from disclosure** Reason for non-disclosure of key personnel details ## **Other optional information** The Cure DHDDS Trustees would like to thank all their donors and volunteers for their support throughout the year. Special mention should go to our DHDDS fundraising heroes during the period: Ian Roberts The Tandem team - Kenny Baillie, Piers Townley, Hamish Clegg, James Keen, Johnnie Wright, Mike Brogan, Matt Goodsall, Ian Roberts, Tim Dixon, Ashley Williams, Matt Lowe, Pete Harris, Nathan and Abi Brown, Will Hamilton, Andrew Paterson, Hugh Yarrow, Ben Peters Katie Barr Sophie Clegg ## **Declarations** **The trustees declare that they have approved the trustees’ report above.** **Signed on behalf of the charity’s trustees** |**Signature(s)**
**Full name(s)**
**Position (eg Secretary,**
**Chair, etc)**
**Date**||| |---|---|---| ||Charles Dixon|Mel Dixon| ||

Trustee|Trustee| ||04/12/2025|| ||04/12/2025|| ||**Cure DHDDS**|**Cure DHDDS**|**Cure DHDDS**|**Cure DHDDS**|**Cure DHDDS**|**No (if any)**|**No (if any)**|**No (if any)**|**CC16a**| |---|---|---|---|---|---|---|---|---|---| ||**For the period**
**from**||01/04/2024||**To**||31/03/2025||| ||||||||||| |**Section A Receipts and payments**|||||||||| |**A1 Receipts**|**Unrestricted**
**funds**
**to the nearest £**
**£127,435.19**
**430**
**-**
**£127,865.30**
**-**
**-**
**-**
**127,865**||**Restricted**
**funds**
**to the nearest £**
**3,630**
**-**
**-**
**3,630**
**-**
**-**
**-**
**3,630**||**Endowment**
**funds**
**to the nearest £**
**-**
**-**
**-**
**-**
**-**
**-**
**-**
**-**||**Total funds**
**to the nearest £**
**131,065**
**430**
**-**
**131,495**
**-**
**-**
**-**
**131,495**||**Last year**
**to the nearest £**| |Donations|**£127,435.19**||||||||**-**| |Interest|**430**||||||||**-**| ||**-**||||||||**-**| |**_Sub total_**_(Gross income for_
_AR)_|**£127,865.30**||||||||**-**| ||||||||||| |**A2 Asset and investment sales,**
**(see table).**|||||||||| ||**-**||||||||| ||**-**||||||||**-**| |**_Sub total_**|**-**||||||||**-**| |**_Total receipts_**
**A3 Payments**|||||||||| ||||||||||**-**| ||||||||||| |Bank Fees|£85.00
£630.98
£4,081.66
£0.00
£171.89
£7,868.00
£480.00
£332.83
£50.00
£21,959.56
£10,000.00
£11,781.21
£8,000.00||||||£85.00
£630.98
£5,686.51
£2,025.00
£171.89
£7,868.00
£480.00
£332.83
£50.00
£21,959.56
£10,000.00
£11,781.21
£8,000.00||**-**| |Computer for Mel|||||||||**-**| |Event Merchandise|||1,605||||||**-**| |Tandem Bike|||2,025||||||**-**| |Website costs|||||||||**-**| |Golf Event|||||||||**-**| |Marathon|||||||||| |Hotel & Travel|||||||||| |Trade Bodymembership|||||||||| |Cardif Grant|||||||||**-**| |UCL|||||||||| |Sainte Justine|||-||-||||**-**| |Birmingham University|||||||||**-**| |**_Sub total_**|**£65,441.13**||**£3,630**||**-**||**69,071**||**-**| ||||||||||| |**A4 Asset and investment**
**purchases, (see table)**|||||||||| ||**-**||||||**-**||| |**_Sub total_ **|**-**||||||**-**||**-**| |**_Total payments_**
**_Net of receipts/(payments)_**
**A5 Transfers between funds**
**A6 Cash funds last year end**
**_Cash funds this year end_**|||||||||| ||||||||||**-**| ||||||||||| ||||||||**62,424**||**-**| ||||||||**-**||**-**| ||||||||**159,238**||**-**| ||**221,662**||||||**221,662**||**-**| CCXX R1 accounts (SS) 10/12/2025 1 |**Section B Statement of assets and liabilities at the end of the period**|**Section B Statement of assets and liabilities at the end of the period**|**Section B Statement of assets and liabilities at the end of the period**|| |---|---|---|---| |**Categories**
Signed by one or two trustees on
behalf of all the trustees
**B5 Liabilities**
**B4 Assets retained for the**
**charity’s own use**
**B3 Investment assets**
**B2 Other monetary assets**
**B1 Cash funds**|Signature
Sainte Justine
Cardiff
Icahn
UCL
Total
**Details**
**Details**
Tandem Bike
**Details**
**_Total cash funds_**
(agree balances with receipts and payments
account(s))
**Details**
**Details**
CAF Account|**Unrestricted**
**funds**
**Restricted**
**funds**
**to nearest £**
**to nearest £**
**221,566**
**-**
**-**
**-**
**-**
**-**
**221,662**
**-**
OK
OK
**Unrestricted**
**funds**
**Restricted**
**funds**
**to nearest £**
**to nearest £**
**-**
**-**
**-**
**-**
**-**
**-**
**-**
**-**
**-**
**-**
**-**
**-**
**Fund to which**
**asset belongs**
**Cost (optional)**
**-**
**-**
**-**
**-**
**-**
**Fund to which**
**asset belongs**
**Cost (optional)**
**-**
**-**
**-**
**-**
**-**
**-**
**-**
**-**
**-**
**Fund to which**
**liability relates**
**Amount due**
**(optional)**
**£11,781.00**
**£23,776.00**
**£35,200.00**
**£80,000.00**
**£150,757.00**
Print Name
Charles Dixon
Melanie Dixon|**Endowment**
**funds**
**to nearest £**| ||||**-**| ||||**-**| ||||**-**| ||||OK| ||||**Endowment**
**funds**
**to nearest £**| ||||**-**| ||||**-**| ||||**-**| ||||**-**| ||||**-**| ||||**-**| ||||**Current value**
**(optional)**| ||||**-**| ||||**-**| ||||**-**| ||||**-**| ||||**-**| ||||**Current value**
**(optional)**| ||||**2,025**| ||||**-**| ||||**-**| ||||**-**| ||||**-**| ||||**-**| ||||**-**| ||||**-**| ||||**-**| ||||**When due**
**(optional)**| ||||| ||||| ||||| ||||| ||||| ||||| ||||Date of
approval| |||Charles Dixon|04/12/2025| |||Melanie Dixon|04/12/2025| CCXX R2 accounts (SS) 10/12/2025 2 ## **Independent examiner's report on the accounts** **Section A Independent Examiner’s Report** ## **Report to the trustees** Charity Name CURE DHDDS **On accounts for the year Charity no** 31 March 2025 **ended (if any)** 1202643 **Set out on pages** (remember to include the page numbers of additional sheets) I report to the trustees on my examination of the accounts of the above charity (“the Trust”) for the year ended **DD / MM / YYYY** 31 March 2025. **Responsibilities and** As the charity's trustees, you are responsible for the preparation of the **basis of report** accounts in accordance with the requirements of the Charities Act 2011 (“the Act”). I report in respect of my examination of the Trust’s accounts carried out under section 145 of the 2011 Act and in carrying out my examination, I have followed all the applicable Directions given by the Charity Commission under section 145(5)(b) of the Act. ------------------------------------------------------------------------------------------- **Independent** [The charity’s gross income exceeded £250,000 and I am qualified to ------------------------------------------------------------------------------------------- **examiner's statement** undertake the examination by being a qualified member of [insert name of ----------------------------applicable listed body]]. _Delete_ [ ] _if not applicable._ I have completed my examination. I confirm that no material matters have --------------------come to my attention in connection with the examination (other than that ---------------------disclosed below *) which gives me cause to believe that in, any material respect: - the accounting records were not kept in accordance with section 130 of the Charities Act; or - the accounts did not accord with the accounting records; or - the accounts did not comply with the applicable requirements concerning the form and content of accounts set out in the Charities (Accounts and Reports) Regulations 2008 other than any requirement that the accounts give a ‘true and fair’ view which is not a matter considered as part of an independent examination. I have no concerns and have come across no other matters in connection with the examination to which attention should be drawn in this report in order to enable a proper understanding of the accounts to be reached. * _Please delete the words in the brackets if they do not apply._ |**Signed:**
**Name:**
**Relevant professional**
**qualification(s) or body**||30/11/2025| |---|---|---| |||| ||Georgia Cragg|| |||| ||ICAEW|| **Oct 2018** 1 **IER** ## **(if any):** **Address:** ## **Section B Disclosure** Only complete if the examiner needs to highlight material matters of concern (see CC32, Independent examination of charity accounts: directions and guidance for examiners). **Give here brief details of any items that the examiner wishes to disclose** . **Oct 2018** 2 **IER**