Experimental DMD drug shows promise in small early trial
NCT ID NCT06079736
First seen Nov 01, 2025 · Last updated May 14, 2026 · Updated 28 times
Summary
This study tested a new drug called PGN-EDO51 in 7 people with Duchenne muscular dystrophy whose genetic mutation can be fixed by skipping a part of their DNA called exon 51. The main goal was to see if the drug is safe and tolerable when given through a vein over several months, with a long-term follow-up. The study was stopped early, so results are limited.
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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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Locations
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British Columbia Children's Hospital
Vancouver, British Columbia, V6H1G9, Canada
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CHU de Québec
Québec, Quebec, G1V4G2, Canada
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Children's Hospital of Eastern Ontario (CHEO)
Ottawa, Ontario, K1H8L1, Canada
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Stan Cassidy Centre for Rehabilitation
Fredericton, New Brunswick, E3B0C7, Canada
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The Hospital for Sick Children (SickKids)
Toronto, Ontario, M5G0A4, Canada
Conditions
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