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 . . .
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 . . .
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 . . .
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 . . .
This year, #GivingTuesday — historically held immediately after the Black Friday and Cyber Monday holiday shopping days following Thanksgiving — is Nov. 27. And this year, MDA is asking our friends to support #MDAGiveStrength, a #GivingTuesday campaign to support kids and adults who need the strength to eat, to breathe, to walk. Muscular dystrophy, ALS . . .
Note: The article in the Journal for Clinical Studies can be found here and begins on P. 60. This is a time of great potential for those living with neuromuscular diseases (NMDs). The pace of progress in science is accelerating and transformative therapies are on the horizon. In just the last few years, the FDA has . . .
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 . . .
When considering a donation to a charitable organization, donors might wonder whether that organization is trustworthy and will use monetary gifts in an effective way to fulfill its mission. With so many different charities requesting donations, and with recent high-profile cases of donor mismanagement and fraud, it is more important than ever for donors to . . .
To learn more about this report, please read our press release. Neuromuscular diseases encompass a broad group of disorders that are individually rare but collectively impact an estimated 250,000 patients in the United States, breaking the rare disease threshold of 200,000 individuals. Currently, treatment options for these diseases are limited. But the financial impact is staggering; . . .
On Oct. 18, pharmaceutical company UCB announced positive results in its phase 2 trial of rozanolixizumab (also known as UCB7665), a potential treatment for myasthenia gravis (MG). Rozanolixizumab, a subcutaneous FcRn (neonatal Fc receptor) monoclonal antibody, performed well in the study, achieving proof-of-concept, improvement in multiple disease-related endpoints and a satisfactory safety profile. The randomized, placebo-controlled, proof-of-concept study enrolled . . .