The Muscular Dystrophy Association (MDA) announced today the awarding of 26 new MDA grants totaling more than $7.5 million toward research focused on a variety of rare neuromuscular diseases
| NEW YORK, May 14, 2019 /PRNewswire/ -- The Muscular Dystrophy Association (MDA) announced today the awarding of 26 new MDA grants totaling more than $7.5 million toward research focused on a variety of rare neuromuscular diseases (NMDs), including Charcot-Marie-Tooth disease (CMT), congenital myopathies, distal myopathy, Duchenne and Becker muscular dystrophies (DBMD), Emery-Dreifuss muscular dystrophy (EDMD), facioscapulohumeral muscular dystrophy (FSHD), Friedreich’s ataxia (FA), giant axonal neuropathy (GAN), inclusion body myopathy (IBM), mitochondrial myopathy, myotonia congenita (MC), myotonic dystrophy (DM), Pompe disease, and spinal muscular atrophy (SMA). This round of grant funding reinforces MDA’s unwavering commitment to the progress of neuromuscular disease research and builds on the more than $1 billion MDA has already invested in research to uncover new treatments and cures for NMDs since its inception.
“MDA awards grants to the world’s best scientists investigating promising theories and therapies that may accelerate treatments and cures for families living with muscular dystrophy, ALS and related neuromuscular diseases,” says Grace Pavlath, PhD, senior vice president and chief research officer at MDA. “Each award will build upon the swift advances that have taken place in research and drug development technologies in the last several years.” Answering crucial questions to speed therapy development The newly funded projects will promote research discoveries and development of new therapies on several fronts. The awarded grants will fund studies that aim to clarify the causes of NMDs, develop new therapeutic approaches, and improve the way clinical trials are conducted. MDA is also committed to maintaining a robust pipeline of promising early-stage scientists who will make the important discoveries of the future. Therefore, this latest round of funding also includes three development grants awarded to investigators at the beginning of their careers and who are on the brink of becoming independent investigators, along with 23 research grants awarded to established, independent investigators. MDA previously announced funding eight new grants this year totaling more than $2 million toward research focused on amyotrophic lateral sclerosis (ALS), a disease in which muscles become weak and eventually nonfunctional. For a complete list of individual awards for this grant cycle, visit MDA’s website and explore the Grants at a Glance section. Highlights from the grant awards for this grant cycle include: Alan Beggs, PhD, professor of Pediatrics at Harvard Medical School and director of the Manton Center for Orphan Disease Research at Children’s Hospital Boston, has been awarded a research grant totaling $300,000 over three years to continue his previous research on the molecular genetics of congenital myopathies. In this project, Dr. Beggs will use whole genome sequencing methods to discover the disease genes and genetic mutations that cause congenital myopathy in patients and families where the underlying cause has not yet been identified. Then, to better understand the biological pathways that lead to disease and search for effective therapies, Dr. Beggs will develop animal models with these mutations to understand how these new mutations lead to disease and screen for new drugs to treat these conditions. It is hoped that this work will have translational and clinical application across many diseases. Kevin Flanigan, MD, attending neurologist at Nationwide Children’s Hospital in Columbus, Ohio, and professor of Pediatrics and Neurology at The Ohio State University College of Medicine, was awarded a clinical trial grant totaling $528,798 over two years to conduct a pilot clinical trial in Duchenne muscular dystrophy (DMD) boys aged 4 to 7 years old to study treatment with spironolactone relative to prednisolone. The mineralocorticoid receptor antagonist spironolactone is an approved drug that has been shown to protect skeletal and cardiac muscle function in DMD mice. Because traditional glucocorticoid steroids like prednisolone can have harmful side effects, this work will be an important step toward finding an alternative treatment by repurposing an already-approved drug. Davide Gabellini, PhD, head of the Gene Expression and Muscular Dystrophy Unit at IRCCS Ospedale San Raffaele in Milan, Italy, was awarded a research grant totaling $297,738 over three years to study the role of Matrin 3 protein (MATR3) in decreasing double homeobox 4 protein (DUX4), the protein known to cause facioscapulohumeral muscular dystrophy. In this study, Dr. Gabellini aims to clarify the DUX4 biological pathway and the mechanism by which DUX4 causes cell death, thereby advancing drug target identification for this disease. All other grant awards for this grant cycle include: Charcot-Marie-Tooth disease (CMT) Charles Abrams, MD, PhD Distal myopathy Aikaterini Kontrogianni-Konstantopoulos, PhD Duchenne and Becker muscular dystrophy (DBMD) Justin Boyer, PhD Diego Fraidenraich, PhD Chad Heatwole, MD M. Carrie Miceli, PhD Foteini Mourkioti, PhD Roger Stromberg, PhD Steven Welc, PhD Emery-Dreifuss muscular dystrophy (EDMD) Tyler Kirby, PhD Facioscapulohumeral muscular dystrophy (FSHD) Jeffrey Miller, PhD Friedreich’s ataxia (FA) Sanjay Bidichandani, PhD Giant axonal neuropathy (GAN) Puneet Opal, MD, PhD Inclusion body myopathy (IBM) Ming Guo, MD, PhD Mitochondrial myopathy Luis Lopez Garcia, PhD Giovanni Manfredi, MD, PhD Luca Scorrano, MD, PhD Myotonia congenita (MC) Mark Rich, MD, PhD Myotonic dystrophy (DM) Thomas Cooper, MD Matthew Disney, PhD Steven Zimmerman, PhD Pompe disease Ellen Roche, PhD Spinal muscular atrophy (SMA) Stephen Meriney, PhD About the Muscular Dystrophy Association MDA is committed to transforming the lives of people affected by muscular dystrophy, ALS and related neuromuscular diseases. We do this through innovations in science and innovations in care. As the largest source of funding for neuromuscular disease research outside of the federal government, MDA has committed more than $1 billion since our inception to accelerate the discovery of therapies and cures. Research we have supported is directly linked to life-changing therapies across multiple neuromuscular diseases. MDA’s MOVR is the first and only data hub that aggregates clinical, genetic and patient reported data for multiple neuromuscular diseases to improve health outcomes and accelerate drug development. MDA supports the largest network of multidisciplinary clinics providing best in class care at more than 150 of the nation’s top medical institutions. Our Resource Center serves the community with one-on-one specialized support, and we offer educational conferences, events, and materials for families and healthcare providers. Each year thousands of children and young adults learn vital life skills and gain independence at summer camp and through recreational programs, at no cost to families. For more information visit mda.org.
SOURCE Muscular Dystrophy Association |
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