Experimental gene therapy tested for rare muscular dystrophy

NCT ID NCT06747273

Terminated Disease control Sponsor: Sarepta Therapeutics, Inc. Source: ClinicalTrials.gov ↗

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.

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.

autosomal recessive limb-girdle muscular dystrophy type 2D limb-girdle muscular dystrophy

As listed by the trial registrant

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

LIMB GIRDLE MUSCULAR DYSTROPHY LIMB GIRDLE MUSCULAR DYSTROPHY TYPE 2D/R3

Contacts and locations

Locations

Children's Hospital of the King's Daughters

Norfolk, Virginia, 23510, United States
Nationwide Children's Hospital

Columbus, Ohio, 43205, United States

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