BetterLife FSHD: a patient-driven health platform
Official title: BetterLife FSHD: A Patient-driven Health and Research Platform
Run by FSHD Society · NCT07409142 · Data updated 2026-11-12
This project tries to connect people with FSHD to personalized resources and collect their health experiences so they can be matched to relevant studies and help improve care.
Quick facts
| Study type | Observational |
|---|---|
| Ages | 1 Year and up |
| Sex | All |
| Enrollment | 5000 (estimated) |
| Sponsor | FSHD Society Academic / other |
| Locations | 1 site (Randolph, Massachusetts) |
| Status | Recruiting |
| Started | August 2, 2024 |
| Main results expected | January 2060 |
| Study ends | January 2060 |
| Registry record updated | February 20, 2026 |
| Trial ID | NCT07409142 on ClinicalTrials.gov |
Is this trial a fit for you?
✓Likely a good fit
Ideal candidates are people aged one year or older who live in the United States or its territories, have a clinical or genetic diagnosis of FSHD or a family history with symptoms, and can provide consent or assent with parental permission as applicable.
✕Probably not a fit
People living outside the United States or its territories or those unwilling to provide consent or share health information are unlikely to benefit from or participate in this platform.
A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.
What this trial studies
BetterLife FSHD is a patient-driven registry that securely collects de-identified health and experience data from people living with facioscapulohumeral muscular dystrophy. Participants complete short surveys on diagnosis, progression, management strategies, and quality-of-life topics on a quarterly and yearly schedule and receive a personalized feed of resources from the FSHD Society. Consent-based data are used to notify participants about clinical trial and research opportunities and to share de-identified datasets with approved researchers, industry, and regulators via a steering committee. Enrollment is open to US residents aged one year and older with a clinical or genetic diagnosis of FSHD or symptomatic individuals with a family history of FSHD.
Why it matters
Potential benefit: If successful, BetterLife could speed identification of eligible participants for trials and give researchers larger real-world datasets to guide improved care and new treatments for FSHD.
How similar studies have gone: Patient registries for rare neuromuscular diseases have previously helped accelerate trial recruitment and natural history research, so the registry approach is established rather than entirely novel.
Who can join: the full rules
Show full inclusion / exclusion criteria
Inclusion Criteria: * Individuals with a clinical or genetic diagnosis of FSHD, or individuals with a family history of FSHD who are showing symptoms * Age 1 year or older * Residing in the United States or its territories * If age 18 or older, individual must be able and willing to provide consent * If under age 18, individual must be able and willing to provide assent, when applicable, and have a parent or legal guardian register and provide consent Exclusion Criteria: * Individuals residing outside the United States or its territories * Unable or unwilling to provide consent, or assent, when applicable
Where it's running
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FSHD Society
Randolph, Massachusetts, United States Recruiting
Study contacts
- Principal investigator: Amanda Hill, MBA — FSHD Society
- Study coordinator: Kayleigh Worek, MS
- Email: betterlife@fshdsociety.org
- Phone: 781-301-6060
Similar studies
Other studies for the same condition or treatment. Recruiting studies are listed first.
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Del-brax (AOC 1020) intravenous treatment for people with FSHD
This study will try regular IV infusions of del-brax (AOC 1020) in adults with FSHD to see if it slows muscle weakness and helps preserve walking ability.
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Apitegromab for FSHD in Adults (Phase 2)
This study tests whether apitegromab works as a standalone treatment for adults with FSHD.
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Assessing respiratory function in FSHD patients using diaphragmatic ultrasound
This study is testing how well diaphragmatic ultrasound can track changes in breathing function over a year in people with Facioscapulohumeral Muscular Dystrophy (FSHD).
-
Safety and early efficacy test of SFL-0821 in adults with FSHD
This study will test whether SFL-0821 is safe and tolerable and whether it shows early signs of benefit in adults with Facioscapulohumeral Muscular Dystrophy (FSHD).
-
Amino acids plus diet and exercise for people with FSHD
This trial will test whether taking essential amino acid supplements together with a structured diet and exercise program helps adults with FSHD build muscle, reduce fat, and improve physical performance compared with placebo and matched healthy adults.
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Umbilical cord lining stem cell treatment for FSHD
This trial will try two intravenous doses of umbilical cord lining–derived stem cells (ULSC) versus placebo to see if they are safe and help people aged 15+ with genetically confirmed FSHD and moderate disease (Ricci score ≥3).
How to take part
- Review the eligibility criteria above with your treating physician.
- Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
- Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.