Gene therapy is linked to better nonmotor outcomes in spinal muscular atrophy type 1 compared to first-line nusinersen, new ...

Nearly all children with spinal muscular atrophy who started on Evrysdi before symptom onset are walking after three years, ...

Imagine a child born in a family with a rare disease, whose treatment would cost more than 17 crore for a single dose. The ...

Court upholds earlier order allowing cheaper SMA drug, calling affordable access more vital than Roche’s monopoly claim.

The division bench upheld the single-judge bench’s findings, which cited public interest in ensuring affordable access to SMA ...

SMA Europe has launched an online tool designed to help patients, doctors, and others in the spinal muscular atrophy (SMA) ...

Kaitlin Batley, MD, a pediatric neurologist at Children’s Health and assistant professor at UT Southwestern Medical Center, discusses how physicians position different therapies for patients with ...

A division bench of Delhi high court Thursday rejected the appeal of pharma giant, Roche, seeking an injunction against ...

The parents of two boys born with a rare genetic disorder have demanded the rollout of a life-changing heel prick test – ...

With this judgment, Natco Pharma can now sell the Spinal Muscular Atrophy treatment drug for ₹15,900 compared to the previous ...