Actively Recruiting
National Registry of Myotonic Dystrophy and Facioscapulohumeral Muscular Dystrophy Patients and Family Members
Led by University of Rochester · Updated on 2025-10-15
3000
Participants Needed
1
Research Sites
N/A
Total Duration
On this page
Sponsors
U
University of Rochester
Lead Sponsor
N
National Institute of Neurological Disorders and Stroke (NINDS)
Collaborating Sponsor
AI-Summary
What this Trial Is About
Researchers are connecting people with myotonic dystrophy (DM) or facioscapulohumeral muscular dystrophy (FSHD), both inherited disorders causing progressive muscle weakness, with scientists studying these diseases. This registry aims to support research and share findings with patients and healthcare providers. Sponsored by the National Institutes of Health, the registry fosters participation and collaboration for these rare muscular dystrophies. To join, individuals can request or download an application containing a Patient Information Form about muscle strength and daily life impact, a Medical Records Release Form for diagnosis verification, and an informed Consent Form explaining the study. Once enrolled, participants can engage in research activities such as completing annual updates on symptom changes and receiving study invitations for questionnaires or treatment explorations. Participation in these activities is voluntary. During the study, researchers collect patient-reported outcomes annually to track disease progression. Participants receive updates on research progress and news through newsletters, emails, and social media. The registry facilitates ongoing communication between patients and researchers without requiring mandatory involvement in further studies. The program has been running since 2000 and will continue through June 2028, with participation duration varying by individual.
CONDITIONS
Brief Title
Myotonic Dystrophy and Facioscapulohumeral Muscular Dystrophy Registry
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Diagnosed with myotonic dystrophy, facioscapulohumeral muscular dystrophy, or related diseases
- Unaffected family member of someone diagnosed with one of these diseases
You will not qualify if you...
History of severe allergic reactions to study medication Currently pregnant or breastfeeding Recent participation in another clinical trial within the last 30 days Presence of uncontrolled medical conditions that could affect safety
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
Duration - Up to several years
Participants provide annual updates to help track how their symptoms change over time and may receive newsletters and study invitations related to muscular dystrophy research.
Annual update form submission
Trial Site Locations
Total: 1 location
1
University of Rochester Medical Center, Department of Neurology
Rochester, New York, United States, 14642
Actively Recruiting
Research Team
R
Registry Coordinator
How is the study designed?
Study Type
OBSERVATIONAL
Masking
N/A
Allocation
N/A
Model
N/A
Primary Purpose
N/A
Number of Arms
0
Similar Trials
Frequently Asked Questions
Have more questions? Get in touch with our team for quick support
Not the Right Trial for You?
Explore thousands of other clinical trials that might be a better match.
Sign up to get personalized trial recommendations delivered to your inbox.
Already have an account? Log in here