Gene editing hope for FSHD patients in early trial
NCT ID NCT06907875
First seen Nov 01, 2025 · Last updated May 15, 2026 · Updated 26 times
Summary
This early-stage study tests a new treatment called EPI-321 for adults with facioscapulohumeral muscular dystrophy (FSHD), a genetic condition that causes muscle weakness. The therapy uses a harmless virus to deliver an epigenetic editing tool that aims to silence the faulty gene. Researchers will monitor 12 participants for safety and any signs that the treatment is working over about 5 years after a single dose.
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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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