Daughter Verena shares her personal story of living with Limb-girdle type 2A muscular dystrophy. The progressive disease affects muscles around the shoulders, limbs, hips, and thighs, making simple ...

Sarepta shares rise more than 12% Analysts see potential for improved perception, commercial uptake Elevidys shows up to 73% reduction in rate of functional decline Jan 26 (Reuters) - Sarepta ...

PHOENIX — Jace Taylor is a rambunctious 4-year-old boy. He runs, jumps and plays with his friends. He isn’t aware that genetically, he’s different from other children. “I think he’s too young to ...

For most new parents, routine postnatal blood testing is a formality. But for some, the test can be life-changing. This was the case for Dino Kong and Emily Kuo, when newborn screening revealed that ...