
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 - Winter 2011
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LEMS - William Atchison, Ph.D.
Grant Announced: Winter 2011Disease Type: Lambert-Eaton Myasthenic Syndrome (LEMS)“Using a mouse model of LEMS, Atchison will explore the means by which certain types of calcium channel proteins are recr...”
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ALS - Ellen Barrett, Ph.D.
Grant Announced: Winter 2011Disease Type: Amyotrophic Lateral Sclerosis (ALS)“These images show motor nerve terminals in the hind limb of a healthy mouse (left) and that of an ALS-affected mouse (ri...”
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Mito. Myopathy - Antoni Barrientos, Ph.D.
Grant Announced: Winter 2011Disease Type: Mitochondrial Myopathies (MM)“Barrientos, pictured with collaborators Flavia Fontanesi and Jingjing Liu, notes that defects in an enzyme called cytoch...”
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DMD/BMD - Harold Bernstein, M.D., Ph.D.
Grant Announced: Winter 2011Disease Type: Duchenne Muscular Dystrophy (DMD)“(Left) A section of mouse leg muscle shows normal muscle fibers, with muscle cell nuclei shown in blue and aligned prope...”
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ALS - James Berry, M.D.
Grant Announced: Winter 2011Disease Type: Amyotrophic Lateral Sclerosis (ALS)“Berry and colleagues are planning a phase 2 human clinical trial of the experimental drug ISIS-333611 as a follow-on stu...”
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ALS - François Berthod, Ph.D.
Grant Announced: Winter 2011Disease Type: Amyotrophic Lateral Sclerosis (ALS)“This image shows mouse nervous system cells cultured in a three-dimensional environment. Motor neurons (red) and Schwann...”
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DMD/BMD - Andrew Brack, Ph.D.
Grant Announced: Winter 2011Disease Type: Duchenne Muscular Dystrophy (DMD)“Brack studies adult muscle stem cells called "satellite cells," and seeks to determine what role, if any, this type of c...”
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FA - Giovanni Coppola, M.D.
Grant Announced: Winter 2011Disease Type: Friedreich's Ataxia (FA)“Because there are numerous compounds with therapeutic potential for Friedreich's ataxia at the preclinical stage of deve...”
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MTM - James Dowling, M.D., Ph.D.
Grant Announced: Winter 2011Disease Type: Endocrine Myopathies“Pictured: (top left) Dowling in his office; (top right) a muscle biopsy sample taken from a person with MTM — the larg...”
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DMD - Dongsheng Duan, Ph.D.
Grant Announced: Winter 2011Disease Type: Duchenne Muscular Dystrophy (DMD)“"Our study will accelerate the tempo of DMD gene therapy research and facilitate its translation to humans with the dise...”
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CMD - Madeleine Durbeej-Hjalt, Ph.D.
Grant Announced: Winter 2011Disease Type: Congenital Muscular Dystrophy (CMD)“The composite image shows: a schematic picture of laminin, the protein that is missing in type 1 congenital muscular dys...”
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DMD - David Goldhamer, Ph.D.
Grant Announced: Winter 2011Disease Type: Duchenne Muscular Dystrophy (DMD)“Goldhamer and colleagues plan to test the hypothesis that adult muscle stem cells called "satellite" cells, known to be ...”
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IBM - Steven Greenberg, M.D.
Grant Announced: Winter 2011Disease Type: Inclusion-Body Myositis (IBM)“Mislocalization of TDP43 protein causes degeneration in the eyes of 1-day-old fruit flies. Greenberg, and co-principal i...”
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LGMD - Michael Hauser, Ph.D.
Grant Announced: Winter 2011Disease Type: Limb-Girdle Muscular Dystrophy (LGMD)“At Duke University, Hauser was part of the team that identified mutations in the myotilin gene (MYOT) that causes LGMD1A...”
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DMD/BMD - Masahiko Hoshijima, M.D., Ph.D.
Grant Announced: Winter 2011Disease Type: Duchenne Muscular Dystrophy (DMD)“An imaging technique called high-resolution Electron Microscope (EM) Tomography provides a 3D rendering of structural he...”
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ALS - Adrian Israelson, Ph.D.
Grant Announced: Winter 2011Disease Type: Amyotrophic Lateral Sclerosis (ALS)“Studies have shown, in spinal cords from the SOD1G37R research mouse model, that there is little accumulation of misfold...”
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LGMD - Peter Kang, M.D.
Grant Announced: Winter 2011Disease Type: Limb-Girdle Muscular Dystrophy (LGMD)“To date, mutations in at least 18 different genes have been found to cause various forms of LGMD, explaining the majorit...”
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DMD/BMD - David Kass, M.D.
Grant Announced: Winter 2011Disease Type: Duchenne Muscular Dystrophy (DMD)“Kass is studying a component of muscle cell membranes called "TRPC" channels which are located in muscle cell membranes....”
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CMD - Shireen Lamande, Ph.D.
Grant Announced: Winter 2011Disease Type: Congenital Muscular Dystrophy (CMD)“Pictured: (Left) image of a collagen 6 microfibril; (Right) unusual collagen 6 aggregates secreted by fibroblasts from a...”
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CNM/MTM - Jocelyn Laporte, Ph.D.
Grant Announced: Winter 2011Disease Type: Endocrine Myopathies“Pictured: Localization of myotubularin (green), the first protein to be implicated in centronuclear myopathy, in culture...”
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MG - Chi Wai Lee
Grant Announced: Winter 2011Disease Type: Myasthenia Gravis (MG)“In the majority of people who develop myasthenia gravis, antibodies attack the acetylcholine receptors (AChR) at the neu...”
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MG - Jon Lindstrom
Grant Announced: Winter 2011Disease Type: Myasthenia Gravis (MG)“Using a rat research model of MG, Lindstrom and colleagues have developed a potential immunosuppressive therapy for the ...”
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DMD/BMD - Jianming Liu, Ph.D.
Grant Announced: Winter 2011Disease Type: Duchenne Muscular Dystrophy (DMD)“A cross-section of skeletal muscle from a research mouse engineered to express high levels of alpha-7 integrin. The dist...”
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MMD - John Lueck, Ph.D.
Grant Announced: Winter 2011Disease Type: Myotonic Dystrophy (DM)“Lueck is seeking the genetic flaws responsible for muscle weakness and degeneration in type 1 myotonic muscular dystroph...”
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MMD - Mani Mahadevan, M.D.
Grant Announced: Winter 2011Disease Type: Myotonic Dystrophy (DM)“"Over the years, many people have asked me, 'What is an RNA foci in MMD1? What RNAs are there and what proteins are part...”
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ALS/SMA - Michael Miller, Ph.D.
Grant Announced: Winter 2011Disease Type: Amyotrophic Lateral Sclerosis (ALS)“Miller and colleagues will use a nematode worm (Caenorhabditis elegans) model to test their central hypothesis. The nema...”
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ALS - Alysson Muotri, Ph.D.
Grant Announced: Winter 2011Disease Type: Amyotrophic Lateral Sclerosis (ALS)“Human neurons derived from induced pluripotent stem cells can be used for modeling neurological diseases in the laborato...”
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CMT - Thien Nguyen, M.D., Ph.D.
Grant Announced: Winter 2011Disease Type: Charcot-Marie-Tooth Disease (CMT)“Nguyen's lab has focused for more than five years on the prevention of axonal degeneration in demyelinating and dysmyeli...”
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DMD - Bradley Olwin, Ph.D.
Grant Announced: Winter 2011Disease Type: Duchenne Muscular Dystrophy (DMD)“Depicted: A mouse muscle that was transplanted and examined after two months, and one that was transplanted and examined...”
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MG - Pragna Patel
Grant Announced: Winter 2011Disease Type: Myasthenia Gravis (MG)“Patel and colleagues are working to identify the gene underlying familial myasthenia gravis in an extremely rare family ...”
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DMD - Pier Lorenzo Puri, M.D., Ph.D.
Grant Announced: Winter 2011Disease Type: Duchenne Muscular Dystrophy (DMD)“Previous MDA support enabled Puri to uncover experimental evidence that treatment with drugs called histone deacetylase ...”
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MMD - Araya Puwanant, M.D.
Grant Announced: Winter 2011Disease Type: Myotonic Dystrophy (DM)“Puwanant's work will help chart the course of myotonic muscular dystrophy over time, which will aid in assessing the eff...”
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MC - Mark Rich, M.D., Ph.D.
Grant Announced: Winter 2011Disease Type: Ion Channel Diseases“Muscle in a dish; Rich's team makes recordings from muscle as part of its efforts to determine whether a single protein ...”
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FA - Joseph Sarsero, Ph.D.
Grant Announced: Winter 2011Disease Type: Friedreich's Ataxia (FA)“"Continued assistance from MDA has made it possible for my research group to make basic laboratory findings and translat...”
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SM - Claudio Sette, Ph.D.
Grant Announced: Winter 2011Disease Type: Spinal Muscular Atrophy (SMA)“Pictured: Neural (nervous system) stem cells grown in conditions that encourage them to mature into motor neurons. The c...”
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DMD - Hao Shi, Ph.D.
Grant Announced: Winter 2011Disease Type: Duchenne Muscular Dystrophy (DMD)“Shi works with a Zeiss confocal laser-scanning microscope, an instrument that generates high-resolution fluorescent imag...”
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ALS - Shanthini Sockanathan, Ph.D.
Grant Announced: Winter 2011Disease Type: Amyotrophic Lateral Sclerosis (ALS)“Sockanathan and colleagues will use a combination of approaches to study the mechanisms underlying regulation, initiatio...”
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DMD - Young-Jin Son, Ph.D.
Grant Announced: Winter 2011Disease Type: Duchenne Muscular Dystrophy (DMD)“Son's research interests include the neuromuscular junction (NMJ) like the ones pictured above, in which axons are stain...”
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SBMA - J. Paul Taylor, M.D., Ph.D.
Grant Announced: Winter 2011Disease Type: Spinal-Bulbar Muscular Atrophy (SBMA)“Taylor recently published study results identifying the specific mechanism by which mutations in the androgen receptor p...”
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ALS - Jiou Wang, M.D., Ph.D.
Grant Announced: Winter 2011Disease Type: Amyotrophic Lateral Sclerosis (ALS)“"We are interested in the molecular mechanisms of motor neuron degeneration conserved from the nematode (a type of worm)...”
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DMD - Jen-Chywan Wang, Ph.D.
Grant Announced: Winter 2011Disease Type: Duchenne Muscular Dystrophy (DMD)“Wang and colleagues are exploring the possibilities of targeting the unwanted side effects that come with glucocorticoid...”
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DMD - Xander Wehrens, M.D., Ph.D.
Grant Announced: Winter 2011Disease Type: Duchenne Muscular Dystrophy (DMD)“In Wehrens' lab, Sameer Ather, M.D., performs echocardiography on research mice with a DMD-like disease. Visible on the ...”
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MD - Xiaohua Wu, M.D., Ph.D.
Grant Announced: Winter 2011Disease Type: Metabolic Myopathies“A snapshot of the genesis of muscle cells in the laboratory. Immature muscle cells called myoblasts (red) fuse together ...”