Leigh Maria Ramos-Platt, MD

The new approval opens the gene therapy to a wider range of children with Duchenne muscular dystrophy, a rare genetic disorder that worsens over time.

Newborn screening found that Greigh had spinal muscular atrophy. He was sent to Children’s Hospital Los Angeles, where gene therapy saved him from paralysis.

Brachial plexus injuries in infants affect the nerves that provide sensation and movement in the shoulder, arm, forearm, hand and fingers. CHLA Experts share what physicians should know about this