In order to develop better methods for capturing the perspective of individuals living with Duchenne muscular dystrophy (DMD) during clinical trials, researchers at the University of California Davis Neuromuscular Research Center are seeking participants for an online survey to monitor movement and daily living abilities. The Duchenne Muscular Dystrophy Life-Time Mobility Scale (DMD-LMS) is a . . .
On September 6th, the U.S. Food and Drug Administration (FDA) announced the approval of TIGLUTIK, an oral suspension form of riluzole for the treatment of amyotrophic lateral sclerosis (ALS). TIGLUTIK, marketed by ITF Pharma, the U.S. subsidiary of Italian specialty pharmaceutical company Italfarmaco, is an oral suspension formulation of riluzole, which has been available in . . .
In honor of Charcot-Marie-Tooth (CMT) Awareness Month, the Muscular Dystrophy Association joins the Charcot-Marie-Tooth Association (CMTA) and the Hereditary Neuropathy Foundation (HNF) to host an externally led patient-focused drug development (PFDD) meeting for CMT. The PFDD meeting is being held on Friday, Sept. 28 at the College Park Marriott Hotel and Conference Center in Hyattsville, . . .
MDA recently announced that it has awarded a research infrastructure grant totaling $550,000 to Jeffrey Rothstein, M.D., Ph.D., Professor of Neurology and Neuroscience and Director, Robert Packard Center for Answer ALS Research, Johns Hopkins University School of Medicine. Answer ALS is a nationwide consortium assembling one of the most comprehensive clinical, genetic, molecular, and biochemical . . .
The Muscular Dystrophy Association is pleased to announce that it is once again partnering with Strength, Science, and Stories of Inspiration (SSSI) to support the second SSSI-MDA fellowship. This fellowship, aimed at graduate students and postdoctoral fellows that already have salary support in their existing labs, will provide additional research funding to allow those individuals to . . .
Late on Wednesday, Sarepta Therapeutics announced that the Food and Drug Administration (FDA) has placed a clinical hold on their Phase 1/2a Duchenne Muscular Dystrophy (DMD) Micro-Dystrophin Gene Therapy Trial due to contamination of the manufacturing lot with plasmid DNA. No safety issues were reported for participants in the trial. Sarepta has stated that they . . .
The Muscular Dystrophy Association recently awarded an MDA clinical research network grant to Nicholas Johnson, vice chair of research and associate professor of neurology at Virginia Commonwealth University in Richmond, to establish the Limb-Girdle Muscular Dystrophy (LGMD) Clinical Research Network. The investment, totaling $700,000 over two years, supports seven centers with expertise in LGMD research . . .
On May 10, Sailormen Inc., one of the largest domestic franchisees of Popeyes Louisiana Kitchen restaurants with 132 locations in five states, announced that its annual MDA Golf Classic and 2018 Appetite for a Cure program raised a record-breaking $1,007,789 for MDA to transform the lives of individuals with muscular dystrophy, ALS and related neuromuscular . . .
Researchers at Orion Pharma are looking for participants with ALS (amyotrophic lateral sclerosis) to participate in the REFALS Phase 3 study. The study is designed to help researchers evaluate the effects of oral levosimendan (ODM-109) on respiratory function in patients with ALS. Participants will be randomized to receive either levosimendan or placebo to determine whether . . .
Researchers at PTC Therapeutics are looking for participants with Duchenne muscular dystrophy to participate in a Phase 3 study. The goal of the study is to evaluate the safety and efficacy of Translarna (ataluren). The drug is believed to promote the formation of full-length and functional dystrophin protein in boys with a nonsense (or premature . . .