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. 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 Vision
Working together as a multi-stakeholder group to expedite FSHD clinical trials to the UK.
Background / History
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. Enter FSHD UK in 2021.
Our Key Aims
Our key aims are to continue to drive for clinical trial readiness for FSHD patients and work as a network to:
- provide information and peer support to FSHD patients, families and carers and in so doing create a mobilized patient community
- raise awareness of FSHD within the wider public, medical and scientific communities.
- support medical and scientific organizations or individuals with activities and projects specifically demonstrating the importance of working as a network and in so doing encourage pharmaceutical companies to bring FSHD clinical trials to the UK
- fund specific research gaps in FSHD.
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.
Who We Are
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).
From a ‘start up’ of 14 people in July 2021 we are now over 30+ people strong. We currently have a network of 6 Clinical sites across the spine of England and 1 ‘guest/observation’ site in Scotland.
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.
Click here to see more about our group.
Our Key Stakeholders
Our external stakeholders include Industry/Regulatory Authorities, the Clinical Trial Research Network (CTRN), European Clinical Network (ECN), FSHD World Alliance, FSHD Europe and more.
We are part of the Global Task Force on Project Mercury which is leading a global initiative on FSHD clinical trial readiness.
To learn more about Project Mercury click here.
