321K views · 2.4K reactions | Today, the Food and Drug Administration approved a new gene therapy for Spinal Muscular Atrophy. SMA Type 1 is the number one genetic killer of children 2 and under. This first-time gene therapy, developed here at Nationwide Children's, is allowing kids like Evelyn to have a bright future. Watch her incredible story. | Nationwide Children's Hospital | Facebook

1K views · 18 reactions | One in 11 thousand babies are born with spinal muscular atrophy or SMA. It's the number one genetic cause of death for infants. But a new gene therapy treatment is saving lives and improving outcomes as explained in this WVU Medicine Health Report | WVU Medicine | Facebook

You may have heard of “gene therapy” or “cell therapy.” But what are they? And how are they different from conventional medicines? | Novartis