The Phase II study of Evrysdi showed significant improvements in survival and motor milestones in babies with type 1 spinal muscular atrophy (SMA).
List view / Grid view
Spinal Muscular Atrophy
New research has found that of the drugs in the pre-registration phase, 10 are expected to become blockbusters over the next six years.
The pharmaceutical company failed to inform the FDA of manipulated data when the gene therapy was under review and could face a legal battle.
A number of drugs currently in their late stages in the development process could see competition for nusinersen, the only current SMA treatment, rise...