Bi-allelic Mutations

Genentech’s Risdiplam on Track for Approval for Spinal Muscular Atrophy

Analysts, Bi-allelic Mutations, Clinical Trials, Drug Pricing, FDA, FDA/Regulatory, Infants, Neuromuscular diseases, New Drug Application (NDA), Potential Blockbusters, R&D, Spinal Muscular Atrophy (SMA), Spinal Muscular Atrophy (SMA), Survival motor neuron 1 (SMN1) gene, Type 1 Spinal Muscular Atrophy (SMA)

Roche company Genentech announced positive top-line results from the pivotal Part 2 of the FIREFISH study looking at risdiplam in infants aged 1-7 months with Type 1 spinal muscular atrophy (SMA).

January 23, 2020/by BioSpace