DMD drug tested in wheelchair users – but trial stops early

NCT ID NCT04708314

Terminated Disease control Sponsor: Rare Disease Research, LLC Source: ClinicalTrials.gov ↗

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.

This is an AI summary of the original study and may miss details. Read our disclaimer.

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Conditions

The condition(s) this trial relates to.

Duchenne muscular dystrophy

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

DUCHENNE MUSCULAR DYSTROPHY

Contacts and locations

Locations

Children's Hospital of Pittsburgh of UPMC

Pittsburgh, Pennsylvania, 15224, United States
Rare Disease Research, LLC

Atlanta, Georgia, 30318, United States

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