FDA Grants Orphan Drug Designation to Myonexus Therapeutics’ MYO-102 Gene Therapy Drug for Limb-Girdle Muscular Dystrophy Type 2D

On Jan. 2, the U.S. Food and Drug Administration (FDA) awarded Orphan Drug Designation for MYO-102, an investigational gene therapy for limb-girdle muscular dystrophy type 2D (LGMD2D), also known as alpha-sarcoglycanopathy. Myonexus Therapeutics has licensed the technology from Nationwide Children’s Hospital, which holds the Investigational New Drug (IND) application for MYO-102. Treatment using gene therapy, . . .

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In Memoriam: Dr. Stephen Katz

We were saddened to end 2018 with the passing of one of the true champions of neuromuscular disease research, Dr. Stephen Katz, the long-time director of the National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS). Dr. Katz led efforts throughout the National Institutes of Health (NIH) to implement the Muscular Dystrophy Community Assistance . . .

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2018 ONEVoice Survey Results Gathered in a New Report

In January 2018, MDA partnered with Edge Research, an established research firm with extensive expertise working with patient advocacy organizations, to conduct an objective and thorough study among the neuromuscular disease community. This survey, a first-of-its-kind for MDA, was called ONEVoice and comprised 50 questions that sought to identify the needs of individuals and families . . .

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Ra Pharmaceuticals Announces Positive Results from Phase 2 Trial of Zilucoplan in Patients with Generalized Myasthenia Gravis

On 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 . . .

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Biogen Announces Positive Phase 1 Results, Plan to License and Develop Ionis Pharmaceuticals’ Investigational Therapy BIIB067 for Familial ALS

On 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 . . .

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Wave Life Sciences Announces Positive Results of Phase 1 Trial of WVE-210201 for Duchenne Muscular Dystrophy

Today, 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 . . .

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FDA Accepts AveXis’ BLA for SMA Drug Zolgensma Under Priority Review

Today, 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 . . .

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Biohaven Pharmaceuticals Announces FDA Has Accepted Its 505(b)(2) New Drug Application to Review BHV-0223 for Treatment of ALS

On Nov. 26, Biohaven Pharmaceuticals announced that the Food and Drug Administration (FDA) has accepted its 505(b)(2) New Drug Application (NDA) to review BHV-0223, a sublingual (placed under the tongue) formulation of riluzole for the treatment of patients living with amyotrophic lateral sclerosis (ALS). In submitting its NDA using the 505(b)(2) pathway, Biohaven was able . . .

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FDA Approves Catalyst Pharmaceuticals’ Firdapse for Treatment of Lambert-Eaton Myasthenic Syndrome

On Nov. 28, the U.S. Food and Drug Administration (FDA) announced the approval of Firdapse (amifampridine phosphate) for the treatment of adults with Lambert-Eaton myasthenic syndrome (LEMS). Firdapse, developed by Catalyst Pharmaceuticals, is an oral potassium channel inhibitor designed to prolong signals released from nerves and allow greater stimulation of muscles. This is the eighth . . .

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Clinical Trial Alert: Stealth BioTherapeutics Seeks Participants for a Phase 3 Mitochondrial Myopathy Trial

Stealth BioTherapeutics is looking for participants with primary mitochondrial myopathy (PMM) to participate in the MMPOWER-3 Phase 3 trial. This trial is designed to evaluate the efficacy and safety of daily subcutaneous injections of elamipretide, an investigational drug that associates with cardiolipin, a key structural component of the inner mitochondrial membrane, and has shown to . . .

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