
Hope Through Research
At MDA, we take a big picture perspective across the full spectrum of neuromuscular diseases to uncover scientific and medical breakthroughs that accelerate treatments and cures. The power in our research approach is that we can often apply learnings from one disease to progress in others to bring urgently-needed answers to our families.
Grants at a Glance - Summer 2016
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LGMD – Michele Calos, Ph.D.
Grant Announced: Summer 2016Disease Type: Limb-Girdle Muscular Dystrophy (LGMD)““We are inspired to study LGMD2B and 2D because we are working with families who have patients with these disorders,...”
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FSHD – Joel Chamberlain, Ph.D.
Grant Announced: Summer 2016Disease Type: Facioscapulohumeral Muscular Dystrophy (FSH, FSHD)““Our area of focus is important in achieving a greater understanding of the early changes in muscle resulting from DUX...”
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OPMD – Anita Corbett, Ph.D.
Grant Announced: Summer 2016Disease Type: Oculopharyngeal Muscular Dystrophy (OPMD)““My specific expertise in the biological area impacted in OPMD was my initial inspiration to study the disease, but no...”
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DMD, LGMD, CMD – Rachelle Crosbie-Watson, Ph.D.
Grant Announced: Summer 2016Disease Type: Duchenne Muscular Dystrophy (DMD)““For the first time, there are numerous ongoing clinical trials focused on Duchenne muscular dystrophy,” Rachelle Cr...”
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SMA – Christine DiDonato, Ph.D.
Grant Announced: Summer 2016Disease Type: Spinal Muscular Atrophy (SMA)“Christine DiDonato aims to characterize the role of SMN protein in skeletal muscle. Her work could lead to development o...”
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ALS – Heather Durham, Ph.D.
Grant Announced: Summer 2016Disease Type: Amyotrophic Lateral Sclerosis (ALS)“Heather Durham’s work focuses on the role of the nBAF protein complex in the health and activity of motor neurons (ner...”
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Mitochondrial Myopathies – Marilena D’Aurelio, Ph.D.
Grant Announced: Summer 2016Disease Type: Mitochondrial Myopathies (MM)““Current treatments for mitochondrial diseases are largely supportive due to the lack of defined targets,” Marilena ...”
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DMD – Diego Fraidenraich, Ph.D., FAHA
Grant Announced: Summer 2016Disease Type: Duchenne Muscular Dystrophy (DMD)““Currently, it is not well understood why certain cells in DMD are more susceptible to damage than others, or how this...”
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ALS – Tania Gendron, Ph.D.
Grant Announced: Summer 2016Disease Type: Amyotrophic Lateral Sclerosis (ALS)““Tremendous progress has been made towards deciphering the underlying causes of ALS, and the recent discovery of the C...”
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IBM and other myopathies – Alfred Goldberg, Ph.D.
Grant Announced: Summer 2016Disease Type: Inclusion-Body Myositis (IBM)““Among the major cellular processes that are dysregulated in muscle disease is protein regulation by the ubiquitin-pro...”
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LGMD and Miyoshi Myopathy – Miranda Grounds, Ph.D.
Grant Announced: Summer 2016Disease Type: Limb-Girdle Muscular Dystrophy (LGMD)““To develop well-informed treatments and cures, we first need to know why the muscle starts to deteriorate,” Miranda...”
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LGMD – Madhuri Hegde, B.S., M.S., Ph.D.
Grant Announced: Summer 2016Disease Type: Limb-Girdle Muscular Dystrophy (LGMD)““Significant advances are being made in finding new therapeutic approaches,” Madhuri Hegde says, “specifically for...”
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FA – Michael Huang, Ph.D.
Grant Announced: Summer 2016Disease Type: Friedreich's Ataxia (FA)““I am passionate about making a difference in the lives of people affected by Friedreich’s ataxia, as I have seen fi...”
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ALS – Christi Kolarcik, Ph.D.
Grant Announced: Summer 2016Disease Type: Amyotrophic Lateral Sclerosis (ALS)““The results from our proposed studies will provide a unique and innovative perspective on the motor neuron degenerati...”
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MG – Linda Kusner, Ph.D.
Grant Announced: Summer 2016Disease Type: Myasthenia Gravis (MG)““This is my first MDA grant,” Linda Kusner says. “Receiving the grant is immensely important to my work. The fundi...”
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DMD – Pura Muñoz-Cánoves, Ph.D.
Grant Announced: Summer 2016Disease Type: Duchenne Muscular Dystrophy (DMD)““DMD patients and families would surely prefer to have a cure for DMD based on targeting the primary defect (loss of d...”
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SMA – Lyndsay Murray, Ph.D.
Grant Announced: Summer 2016Disease Type: Spinal Muscular Atrophy (SMA)““My lab works predominantly on mouse models of spinal muscular atrophy,” Lyndsay Murray says. “It is our goal to u...”
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FA – Marek Napierala, Ph.D.
Grant Announced: Summer 2016Disease Type: Friedreich's Ataxia (FA)““The ultimate goal of this work,” Marek Napierala says, “is to design and conduct proof-of-concept studies of a no...”
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ALS – Leonard Petrucelli, Ph.D.
Grant Announced: Summer 2016Disease Type: Amyotrophic Lateral Sclerosis (ALS)““This is our first MDA grant, and we are honored to be receiving this award,” Leonard Petrucelli says. “At the end...”
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DMD – Pier Lorenzo Puri, M.D., Ph.D.
Grant Announced: Summer 2016Disease Type: Duchenne Muscular Dystrophy (DMD)““I think that the recent progresses in this field that have led to translation into clinical trials of many experiment...”
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Myotonic Dystrophy – Jack Puymirat, M.D., Ph.D.
Grant Announced: Summer 2016Disease Type: Myotonic Dystrophy (DM)““DM1 is one of the muscular dystrophies for which clinical trials are ongoing and several therapeutic approaches are p...”
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ALS – Laura Ranum, Ph.D.
Grant Announced: Summer 2016Disease Type: Amyotrophic Lateral Sclerosis (ALS)““We are using mice to test a novel type of therapy that involves targeting mutant proteins with antibodies,” Laura R...”
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LGMD, IBM and myofibrillar myopathies – Chris Weihl, M.D., Ph.D.
Grant Announced: Summer 2016Disease Type: Limb-Girdle Muscular Dystrophy (LGMD)““My research focuses on how skeletal muscle disposes of and recycles ‘trash’ that accumulates under conditions of ...”
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ALS – Daniela Zarnescu, Ph.D.
Grant Announced: Summer 2016Disease Type: Amyotrophic Lateral Sclerosis (ALS)““Scientifically, I was intrigued by the discovery of RNA defects in ALS and thought this was a great avenue to use my ...”