Survival motor neuron 1 (SMN1) gene Archives

FDA Action Alert: PTC and Roche, Lipocine, Incyte and Morphosys, and BMS

Adults, Babies, Children, Clinical Trials, Complete Response Letter, FDA, FDA/Regulatory, NDA Resubmission, New Drug Applications, PDUFA, Priority Review, R&D, Relapsed Or Refractory (R/R) Large B-Cell Lymphoma (LBCL), Spinal Muscular Atrophy (SMA), Survival motor neuron 1 (SMN1) gene, Survival Motor Neuron 2 (SMN2) Gene

A look at U.S. Food and Drug Administration PDUFA dates for end-of-August and early-September 2020.

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).

Novartis’ SMA Gene Therapy Treatment Approved by FDA, Priced at $2.125 Million

Approvals, Business, Children, FDA, FDA/Regulatory, Gene Therapy, Genetic Diseases, M&A, Pricing, Priority Medicines (PRIME) Designation, Spinal Muscular Atrophy (SMA), Survival motor neuron 1 (SMN1) gene, Therapeutics

The U.S. Food and Drug Administration approved Zolgensma, a gene therapy that Novartis called a life-changing medicine, for children younger than 2 years of age who have been diagnosed with spinal muscular atrophy (SMA) with bi-allelic mutations in the survival motor neuron 1 gene.