Tag: orphan drugs

FDA OKs First ‘Targeted Treatment’ for Rare DMD Mutation

The US Food and Drug Administration (FDA) has approved the antisense oligonucleotide casimersen (Amondys 45, Sarepta Therapeutics) injection for the treatment of patients with Duchenne...

FDA OKs First Oral Treatment for Spinal Muscular Atrophy

The US Food and Drug Administration (FDA) has approved risdiplam (Evrysdi, Roche and PTC Therapeutics) for the treatment of adults and children at least 2...