Solid Biosciences reports that 33 children have now been dosed in their SGT-003 gene therapy trial for Duchenne. So far, the treatment has been generally well tolerated with signs of improved muscle and heart biomarkers. Their global Phase 3 trial is also underway, with the first participant enrolled.
You can advance the care, treatment and cure for Duchenne muscular dystrophy. Contributions in any amount can truly make a difference and can be credited to a fundraiser or event from the list below.
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