MDA announced today the awarding of 25 new MDA grants totaling more than $6.6 million toward research focused on a variety of rare neuromuscular diseases (NMDs), including amyotrophic lateral sclerosis (ALS), Charcot-Marie-Tooth disease (CMT), congenital muscular dystrophy (CMD), Duchenne muscular dystrophy (DMD), facioscapulohumeral muscular dystrophy (FSHD), Friedreich’s ataxia (FA), inclusion body myositis (IBM), limb-girdle muscular dystrophy . . .
This summer MDA challenged our families, friends, sponsors, staff and others around the nation to share their stories about living life without limits despite the challenges of neuromuscular disease. Some shared moments tied to pursuing an education or career that was thought to be out of reach. Some talked about skydiving, hiking or running . . .
Kim Staats, a postdoctoral researcher at the University of Southern California in Los Angeles, was awarded an MDA development grant totaling $180,000 over a period of three years to study potential causes for sporadic amyotrophic lateral sclerosis (ALS). Using a cutting-edge approach to identify genetic contributors in sporadic ALS, Staats has found a new gene . . .
Chris Lorson, professor of veterinary pathology, and molecular biology and immunology at the University of Missouri in Columbia, was awarded an MDA research grant totaling $300,000 over three years to optimize an approach currently in clinical trials for spinal muscular atrophy (SMA) by delivering an enhanced form of drug combined with other potential synergistic therapies. . . .
Kevin Foust, assistant professor in the department of neuroscience at Ohio State University in Columbus, was awarded an MDA research grant totaling $293,378 over three years to investigate disruption of gut bacteria in spinal muscular atrophy (SMA). The gut microbiome is the collection of organisms that inhabit a healthy gastrointestinal (GI) tract. Dysbiosis, or changes . . .
Diane Merry, associate professor at Thomas Jefferson University in Philadelphia, was awarded an MDA research grant totaling $300,000 over three years to identify therapeutic opportunities to promote normal androgen receptor function while preventing the toxic effects of polyglutamine expansion in spinal-bulbar muscular atrophy (SBMA). Working with GlaxoSmithKline, Merry will test small molecule compounds that activate . . .
Colin Crist, an assistant professor in the department of human genetics at McGill University in Québec, Canada, was awarded an MDA research grant totaling $300,000 over three years to investigate a biological mechanism that determines whether skeletal muscle stem cells, which play an important role in muscle regeneration, self-renew or differentiate. Crist has identified inhibitors . . .
Ronald Haller, professor of neurology and neurotherapeutics at University of Texas Southwestern Medical Center in Dallas, was awarded an MDA research grant totaling $300,000 over three years to identify the cause and possible effective treatment for the oxidative defect that accompanies blocked glycogen breakdown in McArdle disease (phosphorylase deficiency). Haller plans to assess levels of . . .