Spinal injection gene therapy trial offers hope for devastating childhood diseases
NCT ID NCT05152823
Summary
This early-phase trial is testing a one-time spinal injection of a gene therapy for children with rare, life-limiting neuromuscular diseases called SMARD1 and CMT2S. The goal is to deliver a working copy of a missing gene to slow or stop disease progression. The study will first check for safety and then see if the treatment can improve motor skills like walking or arm strength.
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Contacts and locations
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Locations
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Nationwide Children's Hospital
Columbus, Ohio, 43205, United States
Conditions
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