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- Ra Pharmaceuticals Announces Positive Results from Phase 2 Trial of Zilucoplan in Patients with Generalized Myasthenia Gravis December 17, 2018On Dec. 10, Ra Pharmaceuticals Inc., based in Cambridge, Mass., announced positive results from a Phase 2 clinical trial designed to evaluate zilucoplan (RA101495) for treating generalized myasthenia gravis (gMG). Patients who received the drug had significant reductions in both measured endpoints, the Quantitative Myasthenia Gravis (QMG) score and the Myasthenia Gravis — Activities of […]
- In “Cinemability,” Director Jenni Gold Asks Hollywood How it Sees Disability December 12, 2018Jenni Gold has been writing, directing and producing film shorts, movies and TV shows for more than 20 years. She’ll tell you, since she was a kid making movies with a Hi8 camcorder, she’s been in love with Hollywood for a lot longer. She’ll also tell you there’s a problem with it. It’s failing the […]
- Biogen Announces Positive Phase 1 Results, Plan to License and Develop Ionis Pharmaceuticals’ Investigational Therapy BIIB067 for Familial ALS December 11, 2018On Dec. 6, Biogen Inc. announced positive Phase 1 clinical trial results and its decision to license and develop partner Ionis Pharmaceuticals’ BIIB067 (IONIS-SOD1RX). BIIB067 is an investigational therapy for treating amyotrophic lateral sclerosis (ALS) caused by superoxide dismutase 1 (SOD1) mutations, a subtype of familial ALS that makes up 2 percent of all ALS […]
- Wave Life Sciences Announces Positive Results of Phase 1 Trial of WVE-210201 for Duchenne Muscular Dystrophy December 6, 2018Today, Wave Life Sciences announced that its Phase 1 trial of WVE-210201, an investigational, exon skipping therapy for boys with Duchenne muscular dystrophy (DMD) amenable to exon 51 skipping, showed positive safety and tolerability data. Based on these results, Wave announced that it will initiate a Phase 2/3 clinical trial of WVE-210201 in 2019. Wave […]
- FDA Accepts AveXis’ BLA for SMA Drug Zolgensma Under Priority Review December 3, 2018Today, Novartis, the parent company of AveXis Inc., announced that the U.S. Food and Drug Administration (FDA) has accepted the company’s Biologics License Application (BLA) under Priority Review for Zolgensma (formerly known as AVXS-101), a gene therapy for the treatment of spinal muscular atrophy (SMA) type 1. Priority Review status requires the FDA to review […]