Experimental gene therapy targets rare muscle disease in first human test
NCT ID NCT05876780
First seen Jun 26, 2026 · Last updated Jun 27, 2026 · Updated 1 time
Summary
This early-phase trial tests a single-dose gene therapy called SRP-9003 in 6 people with limb girdle muscular dystrophy type 2E/R4, a rare genetic muscle-weakening disease. The main goals are to check safety and see if the therapy can produce the missing beta-sarcoglycan protein in muscle. Both ambulatory and non-ambulatory participants are included.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
Active substance
SRP-9003 (a gene therapy given as a single IV infusion)
What this could lead to
If successful, this could point toward a treatment that restores the missing protein in muscle cells, potentially slowing or stopping muscle weakness in LGMD2E/R4.
What could go wrong
This is a very early, small phase 1 trial with only 6 people. It primarily checks safety and protein levels, not whether it improves strength or daily life. Gene therapies can cause immune reactions or other side effects.
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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Nationwide Children's Hospital
Columbus, Ohio, 43205, United States
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St. Jude Children's Research Hospital
Memphis, Tennessee, 38105, United States