New gene therapy trial hopes to silence FSHD at its source
NCT ID NCT06907875
First seen Nov 01, 2025 · Last updated May 23, 2026 · Updated 27 times
Summary
This early-stage trial tests a single-dose gene therapy called EPI-321 in 12 adults with FSHD type 1, a genetic muscle disease. The therapy aims to turn off the faulty gene that causes muscle damage. Researchers will monitor safety and look for early signs that the treatment is working over about 5 years.
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Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
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Contacts and locations
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Study contacts
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Contact
Phone: •••-•••-•••• Email: •••••@•••••
Locations
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David Geffen School of Medicine at University of California, Los Angeles
RECRUITINGLos Angeles, California, 90095, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
Contact
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Kennedy Krieger Institute, Center for Genetic Muscle Disorders
RECRUITINGBaltimore, Maryland, 21205, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
Contact
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Pacific Clinical Research Network
RECRUITINGAuckland, 0622, New Zealand
Contact Phone: •••-•••-•••• Email: •••••@•••••
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Rare Disease Research
RECRUITINGAtlanta, Georgia, 303329, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
Contact
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Royal Alfred Hospital
RECRUITINGSydney, New South Wales, 2050, Australia
Contact
Contact Phone: •••-•••-•••• Email: •••••@•••••
Contact Phone: •••-•••-•••• Email: •••••@•••••
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University of Massachusetts Chan Medical School
RECRUITINGWorcester, Massachusetts, 01605, United States
Contact Email: •••••@•••••
Contact Phone: •••-•••-•••• Email: •••••@•••••
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Utah Program for Inherited Neuromuscular Disorders - University of Utah
RECRUITINGSalt Lake City, Utah, 84112, United States
Contact
Contact Phone: •••-•••-•••• Email: •••••@•••••
Conditions
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