The FDA based its approval of the extended indication for Soliris on comprehensive clinical data from the Phase 3, randomized, double-blind, placebo-controlled, multicenter REGAIN study (ECU-MG-301).
Thymectomy improves clinical outcomes over a three-year period in patients with nonthymomatous myasthenia gravis, according to research published in the Aug. 11 issue of the New England Journal of Medicine.
Impax announced that the Food and Drug Administration (FDA) has approved Pyridostigmine Bromide Extended-Release Tablets, the generic version of Valeant's Mestinon Timespan.
The FDA has granted Breakthrough Therapy designation to drisapersen, an exon-51 skipping compound for the potential treatment of patients with Duchenne Muscular Dystrophy, a lethal form of muscular dystrophy.
Findings in a small group of patients with very low vitamin D levels show that muscle efficiency significantly improves when vitamin D status is improved.
Corticorelin acetate (CrA) administration to patients with peritumoral brain edema (PBE) allows the reduction of steroid doses and is associated with reduced incidence and severity of steroid-induced myopathy and other steroid-related adverse effects.
Milo Biotechnology announced that AAV1-FS344 has been granted Orphan Drug designation from the FDA for the treatment of Becker and Duchenne muscular dystrophy.