Our vision is a world in which all rare diseases have treatments – made together with patients, for patients
How are we achieving our vision?
- We transform the lives of people affected by rare diseases by working directly with patient support groups, giving them the skills to professionalise their work and the confidence to recognise their own expertise in their rare conditions.
- We accelerate change in rare disease research and care by building a strong, supportive, and collaborative rare disease community that works for the benefit of all rare conditions.
What do we do?
Findacure’s projects are in high demand, helping patient groups to:
Build their charity
Raise awareness of their rare condition
Get research ready
Provide support to patients and families
Charity number: 1149646
Company number: 08174973
The need for our organisation was realised out of the separate experiences of our co-founders, Nick Sireau and Tony Hall:
Nick’s two sons were born with the rare disease alkaptonuria, an ultra-rare genetic condition that causes bones to turn black and their surface to corrode. At the time of his sons’ diagnoses, there was no treatment available. Nick quit his job to grow the AKU Society, a charity that now supports patients around the globe and is in the late-stages of running a clinical trial for a promising treatment.
Later in his journey, Nick began to be contacted by other patients and parents wanting to repeat his achievements in their own rare conditions. He realised there was huge demand for a new organisation to share advice and knowledge between patient groups, saving each the time, resources, and stress of having to reinvent the wheel.
As an expert in orphan drug development, Tony had seen first-hand the financial and logistical challenges that pharmaceutical companies face when developing treatments for small patient populations. He realised the need for a non-profit approach to improve patient health while not breaking the NHS bank.
At the time when he met Nick, Tony was interested in establishing an organisation to work on sustainable models for not-for-profit drug development for rare diseases. The two teamed up to establish Findacure and work on his ideas.
Dr. Rick Thompson
Projects and Comms Manager
Mary Rose Roberts
Mary Rose is responsible for delivering and developing Findacure’s events. Primarily, she organises our annual Drug Repurposing for Rare Diseases Conference, the Rare Disease Showcase series and our patient training workshops. Mary Rose also oversees Findacure’s community fundraising events and acts as the charity’s Data Protection Officer. She is keen to encourage communication between industry and patients, and passionate about ensuring Findacure’s activities are engaging and accessible to our community. Mary Rose has a first class hons. degree in Politics from Keele University and joined Findacure in 2016.
Our board of trustees
Dr. Nicolas Sireau
Co-founder and Chairman
Dr. Anthony Hall
Co-founder and Vice-chair
Professor Lakshminarayan R. Ranganath
Our scientific advisory committee
Dr. Bruce Bloom
Dr. Bloom became an Ashoka Social Entrepreneur Fellow for his system-changing solutions to finding new treatments for unsolved diseases. He is the Patient Advisory Board Chair for the Institute for Translational Medicine, the Director of Scientific Affairs for Vision for Tomorrow, on the Science Advisory Boards of Rediscovery Life Sciences, the GARROD AKU Consortium, the Dr. Ralph and Marian Falk Medical Research Trust Awards Programs, and Healx, the Chair of the Governance Committee of the Kendall College Charitable Trust, and a member of the Board of Councilors of Midwestern University, and a member of the editorial board of ASSAY and Drug Development Technologies.
Dr. David Cavalla
He is author of Off-Label Prescribing: Justifying Unapproved Medicine, and one of the first advocates of drug repurposing, using this strategy to create three first-in-class developmental products to have successfully passed human proof of concept clinical stages. He obtained a first degree and PhD at Cambridge University and spent two years as a visiting Fellow at the NIMH, Washington, DC. He is author/inventor of over 70 published papers and patents.
Dr. Mark Edwards
He has also established major R&D projects at the interfaces between academia, the NHS and the biopharmaceutical industry e.g. MRC/industry stratified medicine consortia in COPD and RA, and the NIHR’s Translational Research Partnership Programme.
Now he mainly provides strategic and applied clinical, scientific and policy advice to small and medium-sized biopharmaceutical companies. Mark works with a diverse range of other life sciences organisations at Board/Committee level, or as a business mentor. These include the ME Trust and Dyskeratosis Congenita Action.
Dr. Kathryn Johnson
Kathryn has a long standing interest in neonatal/paediatric rare disease and is committed to supporting research into such conditions with the ultimate aim of improving the lives of babies, children and families. She has a strong research delivery record within the neonatal service and as an executive committee member for the British Paediatric Surveillance Unit has a unique experience and insight into surveillance for paediatric rare disease. Kathryn’s is also Scientific Advisor for the charity Action for Ataxia Telangectasia, where her role is to monitor the progress and outcomes of the research projects the charity funds.
She has found living with a chronic physical illness difficult and challenging. While tolerating the treatment well, the side effects have been overwhelming. She hopes that this is an area where she can give a substantial contribution to the work of the Committee. Repurposing of drugs will be a new field and she is looking forward to meeting the challenges of this important area of work.
Dr. Edmund Jessop
He was Vice President of the UK Faculty of Public Health (FPH) from 2011 to 2014 and Editor of the Journal of Public Health 1998 – 2007. He has taught courses on public health including health economics and health technology assessment for over 15 years.
Dr. Kamlesh Sheth
Our empowerment advisory committee
She has recently been awarded a grant to develop a programme of support designed for patients with lipodystrophy and will be working with the charity Changing Faces and the Foundation of Nursing Studies to do this. Claire has a particular interest in ensuring that healthcare professionals address the mental health and psychological needs of patients with rare disease.
Dr. Jonathan Morton
After graduating from Cardiff University in 2006 with a degree in Pharmacology and a PhD in Medical Biochemistry, Jonathan has spent his entire career working in the rare diseases field. Since 2012 he has held senior leadership positions within the medical communications field and has provided expert guidance to various stakeholders seeking to improve standards of care for patients with rare diseases.
Jonathan specializes in developing innovative, effective and cost-efficient communications strategies to identify and address the key issues that are unique to each rare condition. He has worked across more than 25 therapeutic areas and has supported the development of and access to some of the most innovative treatments for rare diseases in the recent years
An important part of his work has been to support the activities of patient support and advocacy groups. Access to the treatments and care required by patients with rare and difficult conditions has been a high priority.
He is very pleased to be an honorary member of the Turner Syndrome Support Society and a local volunteer representative for the Alzheimer’s society.
People with ataxia have problems with movement, balance and speech. Alan is fighting to return order to the lack of order.
He was proud to receive a nomination from the Bevan Foundation in 2015 in the Health and Wellbeing category. Plus he participated in the Peer mentoring programme, run by Findacure, to progress Atacsia a Fi – Ataxia and Me©, an “on-line” resource for all affected by this with this “Life. Limiting” condition.
Deborah has been involved in the change of status of the drug sodium valproate from The Pharmacovigilance Risk Assessment Committee (PRAC) to the development of the valproate toolkit at the Medicines and Health Product Regulatory Agency (MHRA). This has led her to an involvement in the Patient Group Consultative Forum at the MHRA.