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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MDA CEO Lynn O’Connor Vos Quoted on the Importance of Accountability Benchmarks for Charitable Organizations

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 . . .

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New Report Explains How Big Data and the Therapeutic Pipeline Will be Life Changing for 250,000 People Living with Neuromuscular Disease

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; . . .

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UCB Reports Positive Results in Phase 2 Trial of Rozanolixizumab for Myasthenia Gravis

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 . . .

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AveXis Submits BLA for SMA Gene Therapy AVXS-101 to FDA

This week, AveXis, a Novartis company, reported that it has submitted a Biologics License Application (BLA) to the U.S. Food and Drug Administration (FDA) requesting approval of AVXS-101 to treat infants with spinal muscular atrophy (SMA) type 1. AveXis is also submitting regulatory applications for the therapy in Europe and Japan. If approved, AVXS-101 would . . .

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Pharnext Announces Positive Results from Phase 3 Trial Using PXT3003 to Treat CMT1A

Today, French pharmaceutical company Pharnext SA announced positive topline results from its pivotal Phase 3 clinical trial (PLEO-CMT) evaluating two doses of PXT3003 compared to placebo during 15 months for the treatment of Charcot-Marie-Tooth disease type 1A (CMT1A). PLEO-CMT was a pivotal, 15-month, double-blind Phase 3 study that assessed the efficacy and safety of PXT3003 compared . . .

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Sarepta Therapeutics Announces Positive Updated Results from Gene Therapy Trial for Duchenne Muscular Dystrophy

This week, at the 23rd International Congress of the World Muscle Society in Argentina, Dr. Jerry Mendell of Nationwide Children’s Hospital in Columbus, Ohio, shared additional data relating to Sarepta Therapeutics’ AAVrh74.MHCK7.micro-Dystrophin gene therapy program for Duchenne muscular dystrophy (DMD).  Dr. Mendell had previously presented results for the first three patients in the study back in . . .

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Researchers at SUNY Binghamton Seek Participants for BMD Biomarker and Patient-Reported Outcomes Study

Researchers at the State University of New York at Binghamton’s School of Pharmacy and Pharmaceutical Sciences are seeking participants living with Becker muscular dystrophy (BMD) to participate in a biomarker and patient-reported outcomes study. The purpose of the Becker Muscular Dystrophy Biomarker and Patient-Reported Outcomes Study is to learn more about disease progression in patients . . .

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Genentech Announces New Data for Risdiplam in SMA

On Oct. 3, Genentech announced interim results from the dose-finding parts of the pivotal FIREFISH and SUNFISH studies investigating risdiplam (RG7916) in spinal muscular atrophy (SMA). Risdiplam is an investigational SMN2-splicing modifier that is given by mouth (or g-tube) and distributes widely throughout the body. Risdiplam is designed to help the SMN2 gene produce more . . .

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