Clinical Experience with Gene Therapy in Older Spinal Muscular Atrophy Patients

Onasemnogene abeparvovec was recently approved for the treatment of Spinal Muscular Atrophy (SMA) in children under 2 years; however, clinical trials were primarily completed in children less than 7 months, and so practical experience dosing older children began in summer 2019. Here, we look at the safety and efficacy of onasemnogene in seven infants older than 7 months who were treated at our center.

Source: Pediatric Neurology - February 4, 2021 Category: Neurology Authors: Susan E. Matesanz, Vanessa Battista, Jean Flickinger, Jennifer N. Jones, Elizabeth A. Kichula Source Type: research