Toxicity concerns send Larimar down
The company hopes that its novel approach in Friedreich’s ataxia could still have legs, but other assets are further ahead.
Jury still out on Sarepta’s next generation
The group’s lead next-gen exon skipper beats a low bar set by Exondys, but adverse events loom large.
Zolgensma flatlines, but Spinraza isn’t the beneficiary
A fiercely competitive three-horse race in spinal muscular atrophy is Evrysdi’s for the taking.
Second-quarter catalysts for the smaller players
Key catalysts approach for Orphazyme, Gemini and Immutep.
Gene therapy data shake Solid
Better-looking safety might not be enough to get Solid back in the Duchenne muscular dystrophy game.
Amicus fail dents Pompe pipeline
Amicus follows Sanofi with disappointing pivotal Pompe data, casting further doubt on approvals.
