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 . . .
On Sunday, Nov. 4, chef Dan Churchill crossed the finish line for MDA. The Australian-born, New York-based chef tackled 26 miles in the TCS New York City Marathon representing MDA’s Team Momentum. “This is genuinely a very big privilege and honor for me, on a personal level and giving back,” Dan says. “[The marathon] is . . .
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 . . .
Growing up with Charcot-Marie-Tooth disease (CMT), Lainie Ishbia learned about living with invisible disability — experiencing the daily struggle of movement without looking, outwardly and obviously, as if that’s the case. She also learned, once ankle-foot orthotics (AFOs, or braces) made her disability visible, those devices designed to help make movement easier can also make . . .
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 . . .
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 . . .
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 . . .
Patrick Cusick’s journey with MDA — and with muscular dystrophy — started 12 years ago, in, of all places, a life insurance office. Twenty-nine at the time, the Ohio native and his wife were expecting their first child, and making preparations as parents (or soon-to-be parents) do. Securing life insurance meant taking some medical tests; . . .