Experimental gene therapy tested for rare muscular dystrophy
NCT ID NCT06747273
First seen Jun 26, 2026 · Last updated Jun 26, 2026
Summary
This early-stage trial tested a gene therapy called SRP-9004 in just 4 people with limb girdle muscular dystrophy type 2D/R3, a rare muscle-weakening disease. The main goal was to check safety, not effectiveness. The study was terminated early, 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
SRP-9004 (a gene therapy given by IV infusion)
What this could lead to
If successful, this could point toward a treatment that slows or stops muscle damage in people with this rare form of muscular dystrophy.
What could go wrong
This was a very early (phase 1b) trial with only 4 participants, and it was terminated early. The therapy may not work or could cause side effects. Results are not yet known.
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 the King's Daughters
Norfolk, Virginia, 23510, United States
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Nationwide Children's Hospital
Columbus, Ohio, 43205, United States