Originally published in the Hartford Courant on July 22, 2018. To see the original article, click here. For many years I was consumed by worry and bitterness, thinking I was the only one who was given an unfair lot in life. My body, once athletic and fit, was weakening from the uncompromising progression of an adult-onset neuromuscular disease . . .
As the International Association of Fire Fighters gathers this week in Seattle for its 54th annual convention, we want to recognize and thank this wonderful group of men and women fire fighters who have dedicated countless hours and generosity to the Muscular Dystrophy Association. The IAFF kicked off its 100th anniversary earlier this year. For . . .
This is an unprecedented time for patients living with spinal muscular atrophy (SMA). It has now been almost two years since Spinraza became the first FDA-approved treatment for SMA, and in that time it’s proven to be a transformative therapy. Although there is much more work to be done, Spinraza has made a significant impact . . .
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 . . .