One-Time gene therapy aims to halt rare muscle disease
NCT ID NCT06246513
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This study tests a single dose of SRP-9003 gene therapy in 17 people with limb girdle muscular dystrophy 2E/R4, a genetic muscle-weakening disease. The goal is to restore a missing protein in muscle cells and improve muscle function. Both walkers and non-walkers can join, and the study is now active but not recruiting.
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
-
Children's Hospital of The King's Daughter
Norfolk, Virginia, 23507, United States
-
Fondazione IRCCS Ca Granda Ospedale Maggiore Policlinico
Milan, 20122, Italy
-
Hospital Sant Joan de Deu
Barcelona, 8950, Spain
-
NMRC Gent (UZ Gent)
Ghent, 9000, Belgium
-
Nationwide Childrens Hospital
Columbus, Ohio, 43205, United States
-
Newcastle University
Newcastle upon Tyne, NE1 3BZ, United Kingdom
-
The Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104, United States
-
Universitatsklinikum Essen; Kinderklinik I, Sozialpadiatrisches Zentrum
Essen, North Rhine-Westphalia, 45147, Germany
-
University Hospital Leuven (UZ Leuven)
Leuven, Vlaams Brabant, 3000, Belgium
-
University of California, San Diego-Altman Clinical and Translational Research Institute
La Jolla, California, 92037, United States