The company hopes that its novel approach in Friedreich’s ataxia could still have legs, but other assets are further ahead.
The group’s lead next-gen exon skipper beats a low bar set by Exondys, but adverse events loom large.
A fiercely competitive three-horse race in spinal muscular atrophy is Evrysdi’s for the taking.
Key catalysts approach for Orphazyme, Gemini and Immutep.
Better-looking safety might not be enough to get Solid back in the Duchenne muscular dystrophy game.
Amicus follows Sanofi with disappointing pivotal Pompe data, casting further doubt on approvals.