Researchers at the University of Pittsburgh say a new minimally invasive intervention can target the root cause of neural function loss.

The evolution of functional assessments for spinal muscular atrophy (SMA) highlights the challenges of adapting pediatric ...

The US Food and Drug Administration has approved a new drug application for a tablet version of Evrysdi (risdiplam) for people living with spinal muscular atrophy (SMA).

Spinal muscular atrophy (SMA) is a debilitating genetic condition that’s usually fatal by a few years of age. But an ...

Spinal muscular atrophy (SMA) is a progressive neurodegenerative disorder set in motion before birth. Scientists at St. Jude ...

Exercise can make a significant difference for patients with SMA by working alongside other medical advancements to improve ...

Spinal muscle atrophyorspinal muscular atrophy(SMA) is a genetic disorder that can affect babies, children, and adults. A ...

On this episode, an infant with a harrowing diagnosis defies all odds after receiving a $2 million genetic treatment. Then, a ...

Vincent Gaynor remembers, almost to the minute, when he realized his part in birthing the breakthrough gene therapy Zolgensma ...

Basel: Roche, announced that the U.S. Food and Drug Administration (FDA) has approved a New Drug Application (NDA) for an ...

Roche’s Evrysdi tablet receives US FDA approval for the treatment of spinal muscular atrophy: Basel Friday, February 14, 2025, 13:00 Hrs [IST] Roche, announced that the US Food ...