Day 90 biopsy data reported from first 3 participants dosed in Phase 1/2 INSPIRE DUCHENNE trial —— Average microdystrophin ...

Vincent Gaynor remembers, almost to the minute, when he realized his part in birthing the breakthrough gene therapy Zolgensma ...

An Italian foundation that helped a young boy overcome Wiskott-Aldrich syndrome becomes the first non-profit in the world to ...