An expression of the live unlimited spirit, stories and voices of the MDA community
Sanjay Bidichandani, MBBS, PhD, professor of Pediatrics at the University of Oklahoma Health Sciences Center, has an ongoing MDA research grant (totaling $300,000 over three years) to study the role of epigenetic silencing of the frataxin gene (FXN) in Friedreich’s ataxia (FA). (Epigenetic changes to the genome are factors that influence the activity of genes . . .
Researchers at Italfarmaco are looking for participants with Duchenne muscular dystrophy (DMD) to participate in a phase 3 study. The goal of the study is to evaluate the efficacy and safety of givinostat, a drug that belongs to a class called HDAC inhibitors and is believed to induce the muscle repair process, which may prevent . . .
Receiving a diagnosis of a neuromuscular disease is often not easy — there can be denial, anger, grief, and a frustrating sense that maybe life is, in a way, over. For Wolfgang Pernice, PhD, however, being diagnosed with Charcot Marie Tooth disease (CMT) in his early teens didn’t bother him that much. He says his . . .
Dr. Perry Mansfield at the SENTA Clinic in San Diego, Calif., is looking for healthy individuals and patients with neuromuscular disease who have documented neurologic pharyngeal muscle dysfunction (dysfunction of the voluntary muscles that form the pharynx) to participate in a pilot study. This study will help to prove the diagnostic consistency of transmembrane electromyography . . .
Carlos T. Moraes, PhD, professor of Neurology at the University of Miami’s Miller School of Medicine in Florida, has an ongoing MDA research grant (totaling $300,000 over three years) to develop gene-editing enzymes that can be used to correct genetic mutations causing mitochondrial myopathies. Mitochondrial diseases caused by mutations in the mitochondrial DNA (mtDNA) are most . . .
James Ervasti, PhD, professor of Biochemistry, Molecular Biology, and Biophysics at the University of Minnesota, Twin Cities, was awarded an MDA research grant totaling $300,000 over three years to study the mechanical characterization of dystrophin in Duchenne muscular dystrophy (DMD). DMD is caused by a mutation in the dystrophin gene on the X chromosome that . . .
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 . . .
Stephen Meriney, PhD, professor of Neuroscience and Psychiatry at the University of Pittsburgh, was awarded an MDA research grant totaling $302,587 over three years to develop a combinatorial drug approach to discover possible therapeutics for spinal muscular atrophy (SMA). His work is focused on testing a novel calcium channel agonist (an agonist produces the same . . .
This September, MDA is kicking off 30 Days of Strength, a campaign to raise funds for and awareness of people living with neuromuscular disease. Since the launch of MDA’s annual Labor Day telethon, September has been our most important fundraising period. Across the 30 days of September this year, MDA is sharing stories of the . . .
In May 2019, Jon Olson set out from Astoria, Oregon to bike across the US. He’s dedicating his miles to MDA, the research and care it supports, and the community it — and Jon — represents. So far, he’s raised more than $10,000. He’s ridden more than 2,500 miles and has about 1,000 left to . . .
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