Leigh Maria Ramos-Platt, MD News

A CHLA expert shares her experience of administering this gene therapy for Duchenne—and why comprehensive care is critical for patients.

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

أولا ، اهتزت أصابع يديه. ثم اهتزت أصابع قدميه. الآن، يقوم بتحريك رأسه للجانبين، ويقطب حاجبيه عندما يختلف معك. بالنسبة لعلي المطوع - البالغ من العمر ثلاث أعوام - وبالنسبة لوالديه وأسرته، والأطباء

First, he wiggled his fingers. Then he wiggled his toes. Now, he moves his head side to side, and he furrows his eyebrows when he doesn’t agree with you. For 3-year-old Ali Al-Mutawa—and for his

Twelve-year-old Justin Ybarra likes to joke around, and he likes to have fun. Very rarely does he take life too seriously—even though there have been plenty of things in his life that have been plenty

Everybody who knows 6-year-old Andrew Escamilla always expected he would play professional baseball someday. His father, a diehard Dodgers fan, took the boy to see the Men in Blue at Dodger Stadium

Miguel Torres is a 14-year-old center midfielder with a big dream: to one day play professional soccer. “I love soccer. I like moving the ball around and having the ball at my feet,” the Gardena teen

The Food and Drug Administration has approved the first drug to treat patients with Duchenne muscular dystrophy, the most common muscular dystrophy diagnosed in childhood. Some experts contend that