DMD drug safety study halted early after enrolling just 2 patients
NCT ID NCT04708314
First seen Apr 04, 2026 · Last updated May 15, 2026 · Updated 6 times
Summary
This study looked at the safety of a drug called golodirsen in boys with Duchenne muscular dystrophy (DMD) who can no longer walk. The drug is given weekly through a vein and works by skipping a part of the genetic code to help produce a shorter but functional protein. Only 2 participants were enrolled before the study was stopped early, so the results are very 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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Locations
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Children's Hospital of Pittsburgh of UPMC
Pittsburgh, Pennsylvania, 15224, United States
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Rare Disease Research, LLC
Atlanta, Georgia, 30318, United States
Conditions
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