New drug trial for duchenne MD halted early – what we know
NCT ID NCT06079736
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This phase 2 study tested a drug called PGN-EDO51 in 7 people with Duchenne muscular dystrophy whose genetic mutation can be corrected by skipping exon 51. The drug was given by IV infusion to see if it is safe and tolerable. The trial was terminated, so results are limited.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
Active substance
PGN-EDO51 (a drug given by IV infusion to help produce a shorter but functional dystrophin protein)
What this could lead to
If successful, this could lead to a treatment that slows muscle decline in Duchenne muscular dystrophy patients who are candidates for exon 51 skipping.
What could go wrong
This is an early-phase, small trial (7 participants) focused on safety, not yet on effectiveness. The study was terminated, which raises uncertainty about its future.
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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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