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; . . .
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 . . .
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 . . .
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 . . .