Gene therapy trial offers hope for boys with devastating muscle disease
NCT ID NCT04626674
Summary
This study is testing an experimental gene therapy called SRP-9001 (delandistrogene moxeparvovec) in boys with Duchenne muscular dystrophy (DMD). The main goals are to see if the one-time treatment is safe and if it helps the body produce a crucial muscle protein called dystrophin. The study is enrolling boys from age 2 to young adults, both those who can walk and those who use wheelchairs.
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Contacts and locations
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Study contacts
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Contact
Phone: •••-•••-•••• Email: •••••@•••••
Locations
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Children's Hospital of The King's Daughters
RECRUITINGNorfolk, Virginia, 23507, United States
Contact Email: •••••@•••••
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Nationwide Children's Hospital
RECRUITINGColumbus, Ohio, 43205, United States
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Stanford University
RECRUITINGPalo Alto, California, 94304, United States
Contact Email: •••••@•••••
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University of California, Davis
RECRUITINGSacramento, California, 95616, United States
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Washington University in St. Louis
RECRUITINGSt Louis, Missouri, 21205, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
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Conditions
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