DMD drug tested in wheelchair users – but trial stops early
NCT ID NCT04708314
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This study tested the safety of golodirsen (Vyondys 53) in boys and men with Duchenne muscular dystrophy who can no longer walk. Only 2 people took part before the trial was stopped early. Participants received weekly IV infusions for up to 96 weeks, with extra follow-up. The goal was to check for side effects and see how the drug affects breathing and arm function.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
Active substance
golodirsen (Vyondys 53)
What this could lead to
If successful, this could help confirm that golodirsen is safe for non-ambulant DMD patients, potentially expanding treatment access to those who can no longer walk.
What could go wrong
The trial was terminated early and enrolled only 2 people, so results are very limited. It is unclear if golodirsen provides meaningful benefit in this advanced stage of the disease.
Disclaimer
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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.
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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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
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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