The first standards of care for Duchenne muscular dystrophy (DMD) were published by the U.S. Centers for Disease Control and Prevention (CDC) in 2010. And today — on World Duchenne Awareness Day 2018 — the DMD care landscape is already, and excitingly, a much different space. Several new therapies, including the first-ever disease modifying DMD . . .
In the last few years, the Duchenne muscular dystrophy (DMD) therapeutic landscape has undergone a dramatic evolution. More therapies are in clinical development today than ever before, each one aiming to treat DMD in a different way in order to halt or slow the disease’s devastating effects. This September marks the two-year anniversary of the . . .
The future of Charcot-Marie-Tooth research and treatments rests where the disease begins: in genes. While many new genes involved in CMT have been identified, we know as many as 40 percent of people living with CMT still don’t have a confirmed genetic diagnosis. We’re also still trying to understand the disease mechanisms of the genes . . .
Today, MDA announced the award of 34 new grants totaling $9.9 million for its Summer 2018 grant cycle. These new grants represent a continued commitment by MDA to fund groundbreaking research that will one day lead to treatments and cures for the diseases in its program. Each grant will impact neuromuscular disease research in a . . .
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 . . .