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2025-03-31-accounts

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:

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.

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