The UK Facioscapulohumeral Muscular Dystrophy Patient Registry
Status: Recruiting
Location: See location...
Intervention Type: Other
Study Type: Observational
SUMMARY
Facioscapulohumeral Dystrophy (FSHD) is the third most common form of neuromuscular dystrophy worldwide with an estimated prevalence of one in 20,000. FSHD is an autosomal dominant genetic disease and is estimated to affect up to 3,000 people in the UK. The patient registry facilitates a questionnaire based research study to better characterise and understand the disease in the UK, and helps to identify potential participants eligible for clinical trials.
Eligibility
Participation Requirements
Sex: All
Healthy Volunteers: f
View:
• \- All patients with a confirmed FSHD diagnosis (or pending diagnosis) who reside in the UK are eligible for inclusion.
Locations
Other Locations
United Kingdom
John Walton Muscular Dystrophy Research Centre
RECRUITING
Newcastle Upon Tyne
Contact Information
Primary
Registry Project Manager and Curator
helen.walker2@newcastle.ac.uk
0191 2418640
Backup
Registries Team
registries@ncl.ac.uk
Time Frame
Start Date: 2013-05
Estimated Completion Date: 2040-01
Participants
Target number of participants: 1018
Treatments
Participants with FSHD
Patients with a confirmed or pending diagnosis of FSHD, living in the UK are eligible to join the registry. Parents/guardians can register a child under 16 years old.
Related Therapeutic Areas
Sponsors
Leads: Newcastle University