Experimental gene therapy for rare muscle disease tested in just 2 people
NCT ID NCT05906251
First seen Jun 26, 2026 · Last updated Jun 26, 2026
Summary
This was a very early (Phase 1) study testing a gene therapy called SRP-6004 for people with limb girdle muscular dystrophy type 2B/R2, a rare muscle-weakening disease. The goal was to see if a single IV infusion of the therapy is safe and can help the body produce a missing protein called dysferlin. Only 2 participants were enrolled, and the study was terminated early, so we have very limited information about its effects.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
Active substance
SRP-6004 (a gene therapy given as a single IV infusion)
What this could lead to
If successful, this could point toward a treatment that restores dysferlin protein in muscle cells, potentially slowing or stopping muscle weakness in LGMD2B/R2.
What could go wrong
This was a very early (Phase 1) trial with only 2 participants and was terminated, so we don't know if it works or is safe. Gene therapies can cause immune reactions or other serious side effects.
Disclaimer
Read more
Show less
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.
Get updates
Get notified about this study
Sign up to get updates when this study changes or when new studies for LIMB GIRDLE MUSCULAR DYSTROPHY are added.
By submitting, you agree to our Terms of use
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
Show contact details
Enter your email to view the contact information for this study.
By submitting, you agree to our Terms of use
Locations
-
Nationwide Children's Hospital
Columbus, Ohio, 43205, United States