
Innovations in Science
We're accelerating the delivery of treatments and cures.
Winter Grants 2019
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CMT - Charles Abrams, MD, PhD
Funded: 02/01/19 through 01/31/22Grant Announced: Winter 2019Disease Type: Charcot-Marie-Tooth Disease (CMT)“Unraveling the cause of a human disease requires having appropriate animal models for the disease being studied. This pr...”
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ALS - Samuel Alworth, MS, MBA
Funded: 10/23/18 through 10/31/20Grant Announced: Winter 2019Disease Type: Amyotrophic Lateral Sclerosis (ALS)
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Congenital & Other Myopathies – Alan Beggs, PhD
Funded: 02/01/19 through 01/31/22Grant Announced: Winter 2019Disease Type: Congenital Myopathies“Alan Beggs, PhD, professor of Pediatrics at Harvard Medical School and director of the Manton Center for Orphan Disease ...”
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FA - Sanjay Bidichandani, PhD
Funded: 02/01/19 through 01/31/22Grant Announced: Winter 2019Disease Type: Friedreich's Ataxia (FA)“The mechanism of gene silencing in FA is known as epigenetic silencing. As we dig deeper, we realize that the silencing ...”
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DMD - Justin Boyer, PhD
Funded: 02/01/19 through 01/31/22Grant Announced: Winter 2019Disease Type: Duchenne Muscular Dystrophy (DMD)“Our research will help define the role of satellite cells in muscular dystrophy. The findings from our proposed work wil...”
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DM - Tom Cooper, MD
Funded: 02/01/19 through 01/31/22Grant Announced: Winter 2019Disease Type: Myotonic Dystrophy (DM)“The overall goal of the work is to understand the molecular and physiological pathways leading from the disease-causing ...”
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DM - Matthew Disney, PhD
Funded: 02/01/19 through 01/31/22Grant Announced: Winter 2019Disease Type: Myotonic Dystrophy (DM)“We hope to establish a new paradigm in drug design in which one can target RNA with small molecules and use small molecu...”
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DMD - Kevin Flanigan, MD, PhD
Funded: 10/05/18 through 10/04/20Grant Announced: Winter 2019Disease Type: Duchenne Muscular Dystrophy (DMD)
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DMD - Diego Fraidenraich, PhD
Funded: 02/01/19 through 01/31/22Grant Announced: Winter 2019Disease Type: Duchenne Muscular Dystrophy (DMD)“A combination of pharmacological treatment that involves Cx43 peptide mimetics and gene editing in phosphorylation sites...”
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FSHD - Davide Gabellini, PhD
Funded: 02/01/19 through 01/31/22Grant Announced: Winter 2019Disease Type: Facioscapulohumeral Muscular Dystrophy (FSH, FSHD)“With our work, we hope to better understand how DUX4 is regulated and how we could control aberrant DUX4 in FSHD for the...”
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ALS - Thomas Gaj, PhD
Funded: 02/01/19 through 01/31/22Grant Announced: Winter 2019Disease Type: Amyotrophic Lateral Sclerosis (ALS)“We’re in the middle of a scientific revolution that’s seeing the development of increasingly precise and effective t...”
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Mitochondrial Myopathy - Luis Lopez Garcia, PhD
Funded: 02/01/19 through 01/31/22Grant Announced: Winter 2019Disease Type: Mitochondrial Myopathies (MM)“We expect to learn more about the CoQ metabolism in health and disease, including regulatory mechanisms of CoQ biosynthe...”
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ALS - Tania Gendron, PhD
Funded: 02/01/19 through 01/31/22Grant Announced: Winter 2019Disease Type: Amyotrophic Lateral Sclerosis (ALS)“We hope that the biomarkers of cognitive impairment identified through this work will prove useful in the clinical asses...”
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ALS - Jonathan Glass, MD
Funded: 11/01/18 through 10/31/21Grant Announced: Winter 2019Disease Type: Amyotrophic Lateral Sclerosis (ALS)“Research into ALS cannot move forward without the participation of patients, who provide the needed clinical information...”
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ALS - Junjie Guo, PhD
Funded: 02/01/19 through 01/31/22Grant Announced: Winter 2019Disease Type: Amyotrophic Lateral Sclerosis (ALS)“The pathophysiological mechanisms underlying ALS and other neuromuscular diseases are still poorly understood. Only thro...”
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IBM - Ming Guo, MD, PhD
Funded: 02/01/19 through 01/31/22Grant Announced: Winter 2019Disease Type: Inclusion-Body Myositis (IBM)
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DMD - Chad Heatwole, MD
Funded: 10/15/18 through 09/30/20Grant Announced: Winter 2019Disease Type: Duchenne Muscular Dystrophy (DMD)“At the completion of our work the DMD research community will have multiple validated outcome measures to aid in therape...”
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EDMD - Tyler Kirby, PhD
Funded: 02/01/19 through 01/31/22Grant Announced: Winter 2019Disease Type: Emery-Dreifuss Muscular Dystrophy (EDMD)“If we determine that DNA damage is a major cause of developing this form of muscular dystrophy, this will open up a new ...”
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CMT - Kleopas Kleopa, MD
Funded: 02/01/19 through 01/31/22Grant Announced: Winter 2019Disease Type: Charcot-Marie-Tooth Disease (CMT)“By establishing an effective, clinically applicable gene therapy approach targeting peripheral nerves, we may be able to...”
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ALS - Csaba Konrad, PhD
Funded: 02/01/19 through 01/31/22Grant Announced: Winter 2019Disease Type: Amyotrophic Lateral Sclerosis (ALS)“We believe finding biomarkers that stratify sporadic ALS patients into subgroups with shared cellular pathology may be t...”
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Distal Myopathy - Aikaterini Kontrogianni-Konstantopoulos, PhD
Funded: 02/01/19 through 01/31/22Grant Announced: Winter 2019Disease Type: Distal Myopathies“Although mutations in MyBP-C1 have been previously linked to severe and lethal forms of distal arthrogryposis, prior to ...”
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ALS - John Landers, PhD
Funded: 02/01/19 through 01/31/22Grant Announced: Winter 2019Disease Type: Amyotrophic Lateral Sclerosis (ALS)“I hope that we will identify several genes that, when inactivated, will lead to a beneficial effect in several models of...”
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Mitochondrial Myopathy/ALS - Giovanni Manfredi, MD, PhD
Funded: 02/01/19 through 01/31/22Grant Announced: Winter 2019Disease Type: Amyotrophic Lateral Sclerosis (ALS)“CHCHD10 mutations are relatively rare causes of neuromuscular disease, but the disease mechanisms and the therapeutic ap...”
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ALS - Ze’ev Melamed, PhD
Funded: 02/01/19 through 01/31/22Grant Announced: Winter 2019Disease Type: Amyotrophic Lateral Sclerosis (ALS)“I believe that my proposed study may open the door to a completely new and promising direction in ALS research. Implemen...”
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SMA - Stephen Meriney, PhD
Funded: 02/01/19 through 01/31/22Grant Announced: Winter 2019Disease Type: Spinal Muscular Atrophy (SMA)“This project will directly test a novel treatment to address neuromuscular weakness in SMA patients. This treatment is n...”
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DMD - M. Carrie Miceli, PhD
Funded: 02/01/19 through 01/31/22Grant Announced: Winter 2019Disease Type: Duchenne Muscular Dystrophy (DMD)“Identification of immune populations involved in DMD pathogenesis may aid in identifying therapeutic targets. Assessin...”
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FSHD - Jeffrey Miller, PhD
Funded: 02/01/19 through 01/31/22Grant Announced: Winter 2019Disease Type: Facioscapulohumeral Muscular Dystrophy (FSH, FSHD)“We will attempt to identify drugs that prevent DUX4-induced changes in mRNA handling proteins and then determine, in mod...”
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DMD - Foteini Mourkioti, PhD
Funded: 02/01/19 through 01/31/22Grant Announced: Winter 2019Disease Type: Duchenne Muscular Dystrophy (DMD)“Knowledge gained from this MDA funded studies will reveal new mechanisms of cardiac dystrophy, providing fresh strategie...”
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GAN - Puneet Opal, MD, PhD
Funded: 02/01/19 through 01/31/22Grant Announced: Winter 2019Disease Type: Giant Axonal Neuropathy (GAN)“While GAN is rare, neurofilament accumulation is seen in a wide range of neurodegenerative disorders, including ALS, Par...”
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MC - Mark Rich, MD, PhD
Funded: 02/01/19 through 01/31/22Grant Announced: Winter 2019Disease Type: Myotonia Congenita (Thomsen Disease and Becker Type)“More recently we have discovered other currents in skeletal muscle that we are currently characterizing in mouse models ...”
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Pompe Disease – Ellen Roche, PhD
Funded: 10/01/18 through 09/30/21Grant Announced: Winter 2019Disease Type: Metabolic Myopathies
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Mitochondrial Myopathy - Luca Scorrano, MD, PhD
Funded: 02/01/19 through 01/31/22Grant Announced: Winter 2019Disease Type: Mitochondrial Myopathies (MM)“We expect to provide a proof of principle that we can curtail the progression of mitochondrial myopathies by acting not ...”
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DMD - Roger Stromberg, PhD
Funded: 02/01/19 through 01/31/21Grant Announced: Winter 2019Disease Type: Duchenne Muscular Dystrophy (DMD)“Hopefully more compounds will be developed (to benefit more patients) and better compounds will be developed (to benefit...”
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ALS - Marka Van Blitterswijk, MD, PhD
Funded: 02/01/19 through 01/31/22Grant Announced: Winter 2019Disease Type: Amyotrophic Lateral Sclerosis (ALS)“By increasing our understanding of this debilitating disease, especially its causes, I am optimistic that we might be ab...”
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DMD - Steven Welc, PhD
Funded: 02/01/19 through 01/31/22Grant Announced: Winter 2019Disease Type: Duchenne Muscular Dystrophy (DMD)“Identifying processes that regulate muscle fibrosis and inflammation are clinically important and could lead to novel st...”
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DM - Steven Zimmerman, PhD
Funded: 02/01/19 through 01/31/22Grant Announced: Winter 2019Disease Type: Myotonic Dystrophy (DM)“There is a lot known about the molecular origin of the disease and this has provided some very clear targets. What is ne...”