About Us
Why We Began
The UK has an excellent reputation for high quality FSHD clinical services, research teams and the established UK FSHD Patient Registry. These accomplished UK centres were mainly working in their own areas without an overarching strategy or central co-ordination. There was no single group focused strategically on FSHD. The UK had missed important opportunities for clinical trials in 2020. There was a gap in making things happen for FSHD in the UK.
How We Began
FSHD UK was set up in July 2021 by Rajeshri Badiani with the aim of expediting clinical trials for patients in the UK. It has since become the recognized group for coordinating such FSHD activities in the UK.
Where We Are Today
We have become a strong multi-stakeholder network and have had a huge success in fast-tracking FSHD activities in the UK. In December 2023 we formed a registered charity to continue to deliver on our vision and aims.
Our Collaborators
“FSHD UK stands at the forefront of driving faster progress in treatment development and access for individuals and families living with FSHD across the UK and around the world.”
– Mark Stone, FSHD Society
What is FSHD?
Facioscapulohumeral muscular dystrophy (FSHD) is a disorder characterized by muscle weakness and wasting (atrophy). The condition gets its name from the muscles most commonly affected:
- face (facio)
- shoulder blades (scapulo)
- upper arms (humeral)
Some of the symptoms are shown in the diagram provided. For a fuller understanding, please refer to the FSHD Society link below.
Information for…
Patients
Our patient section points to key information sources and support organisations that may help you on your FSHD journey. You will find pointers to other FSHD Organisations; UK FSHD Registry, FSHD Society, MDUK and more.
Clinicians
Our clinician section provides up-to-date information on clinical trials and natural history studies on FSHD. There are links to guidance on clinical trial processes and natural history studies. We also introduce the clinicians that are part of the FSHD UK network who are currently running these studies in the UK.
Researchers
In our research section we will share basic research papers/information shared with us by researchers on FSHD around the world. We hope to update our patient community on up and coming developments in FSHD.
Our Key Aims
Our key aims are to continue to drive for clinical trial readiness for FSHD patients and work as a network to:
Aim 1: Information and Support
Provide information and peer support to patients, families, and carers to build a mobilized community
Aim 2: Raising Awareness
Raise awareness of FSHD among the public, medical, and scientific communities
Aim 3: Collaboration
Attract FSHD clinical trials to the UK by fostering collaboration with researchers and industry
Aim 4: Funding
Fund research to address critical gaps in FSHD knowledge
From mid-2023 FSHD Clinical Trials have started in the UK.
Our work does not stop here.
We have more to do to make sure we get up and coming clinical trials to the UK, access to appropriate therapies, and that research into FSHD does not stop.
Our Team
We work with a group of stakeholders that include clinicians, clinical support teams, patients, UK FSHD Registry, research scientists, MDUK, FSHD Society and the Muscular Dystrophy Muscle Centre (Midlands).
This stakeholder model and our ways of working with our five teams overarched by a Core Team has been a bedrock to our success and has enabled us to drive multiple initiatives.
Sub-Team 1
Stakeholder Management, Health Economics Review, and Fundraising
Sub-Team 2
Clinical Site Readiness and In-Clinic Assessments
Sub-Team 3
Patient Engagement
Sub-Team 4
Research Strategy
Sub-Team 5
Technical / Web / IT
