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Grants at a Glance — Winter 2011

  • Ellen Barrett (ALS)

    ALS — Barrett

    Ellen Barrett, professor of physiology and biophysics at the University of Miami (Florida) Miller School of Medicine was awarded an MDA grant totaling $297,102 over three years. The funds will support Barrett's study of the disease process and potential therapies in familial, or inherited, ALS (amyotrophic lateral sclerosis, or Lou Gehrig's disease).

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  • James Berry (ALS)

    ALS — Berry

    MDA has awarded a clinical research training grant totaling $180,000 to clinical research fellow James Berry at Massachusetts General Hospital (MGH) in Boston. The grant will support completion of a two-year fellowship during which Berry plans to study the effects of a drug called ISIS-333611 in familial, or inherited, ALS (amyotrophic lateral sclerosis, or Lou Gehrig’s disease).

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  • François Berthod (ALS): This image shows mouse nervous system cells cultured in a three-dimensional environment. Motor neurons (red) and Schwann cells (green) are positioned at the top of the model, or what would be considered the "spinal cord compartment." The long axons extending from these cells can be seen migrating down through the bottom part of the model, which mimics connective tissue. Cell nuclei are stained blue.

    ALS — Berthod

    MDA has awarded a grant totaling $347,094 over three years to François Berthod, a professor in the department of surgery at Laval University in Quebec City, Quebec, Canada. The funds will help support Berthod's study of the underlying molecular mechanisms and disease process in ALS (amyotrophic lateral sclerosis, or Lou Gehrig's disease).

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  • Adrian Israelson (ALS): Studies have shown, in spinal cords from the SOD1G37R research mouse model, that there is little accumulation of misfolded SOD1 at disease onset, but that it preferentially locates within motor neurons.

    ALS — Israelson

    MDA has awarded a research development grant totaling $180,000 over three years to Adrian Israelson, a postdoctoral researcher at the University of California, San Diego in La Jolla, Calif. The funds will help support Israelson’s study of the underlying mechanisms governing motor neuron (nerve cell) death in SOD1-related familial ALS (amyotrophic lateral sclerosis, or Lou Gehrig's disease).

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  • Alysson Muotri (ALS): Human neurons derived from induced pluripotent stem cells can be used for modeling neurological diseases in the laboratory. Pictured are two neurospheres, compact masses of immature neuron "precursor" cells, derived from human pluripotent stem cells, as captured by a fluorescent microscope. Differentiated (mature) neurons, whose nuclei are shown in red, have begun to extend neuronal processes (green) toward one another, forming neuronal connections. (Artwork by M. Marchetto/A. Muotri

    ALS — Muotri

    MDA has awarded a research grant totaling $362,466 over three years to Alysson Muotri, assistant professor at the University of California, San Diego in La Jolla. The new funds will help support Muotri’s generation of a new research model of ALS (amyotrophic lateral sclerosis, or Lou Gehrig’s disease).

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  • Shanthini Sockanathan (ALS): Sockanathan and colleagues will use a combination of approaches to study the mechanisms underlying regulation, initiation, extent and rate of nerve cell maturation.

    ALS — Sockanathan

    MDA has awarded a research grant totaling $396,000 over three years to Shanthini Sockanathan, associate professor of neuroscience at Johns Hopkins School of Medicine in Baltimore. The new funds will help support Sockanathan’s study of motor neuron (nerve cell) development in neurodegenerative diseases such as ALS (amyotrophic lateral sclerosis, or Lou Gehrig’s disease).

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  • Jiou Wang (ALS)

    ALS — Wang

    MDA has awarded a research grant totaling $330,000 over three years to Jiou Wang, assistant professor of biochemistry & molecular biology and neuroscience at Johns Hopkins University in Baltimore. The new funds will help support Wang’s study of the molecular mechanisms underlying ALS (amyotrophic lateral sclerosis, or Lou Gehrig’s disease).

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  • Michael Miller (ALS/SMA)

    ALS/SMA — Miller

    MDA has awarded a research grant totaling $350,133 over three years to Michael Miller, associate professor in the department of biology at the University of Alabama, Birmingham. The grant will help support Miller's research into the fundamental molecular mechanisms responsible for the death of nerve cells called motor neurons in ALS (amyotrophic lateral sclerosis, or Lou Gehrig's disease) and spinal muscular atrophy (SMA).

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  • Madeleine Durbeej-Hjalt (CMD): The composite image shows: a schematic picture of laminin, the protein that is missing in type 1 congenital muscular dystrophy; Durbeej-Hjalt; and laminin alpha 2 deficient mice treated with a proteasome inhibitor called MG-132 (right) and an untreated mouse (left).

    CMD — Durbeej-Hjalt

    MDA has awarded a research grant totaling $442,023 over three years to Madeleine Durbeej-Hjalt, a professor in muscle biology at Lund University (Sweden), Department of Experimental Medical Science. The grant will help support Durbeej-Hjalt's study of muscle-protein degradation processes in type 1A congenital muscular dystrophy (CMD1A).

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  • Shireen Lamande (CMD): (Left) image of a collagen 6 microfibril; (Right) unusual collagen 6 aggregates secreted by fibroblasts from a CMD patient who does not have a collagen 6 mutation. The mutation in this patient, Lamande says, might be in the gene for a protein that interacts with collagen 6.

    CMD — Lamande

    MDA has awarded a research grant totaling $251,596 over two years to Shireen Lamande, senior research fellow and group leader for muscular dystrophy research and musculoskeletal disorders at Murdoch Childrens Research Institute in Parkville, Victoria, Australia. The new funds will help support Lamande’s research into the identification of new genes responsible for two types of congenital muscular dystrophy (CMD), Bethlem myopathy and Ullrich congenital muscular dystrophy.

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  • Thien Nguyen (CMT)

    CMT — Nguyen

    MDA has awarded a research grant totaling $420,000 over three years to Thien Nguyen, assistant professor in the department of neurology at Johns Hopkins University School of Medicine in Baltimore. The new funds will help support Nguyen’s research into the breakdown of peripheral nerves (nervous tissue that connects the spinal cord with muscles and sensory organs) in Charcot-Marie-Tooth disease (CMT).

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  • Jocelyn Laporte (CNM/MTM): Pictured a localization of myotubularin (green), the first protein to be implicated in centronuclear myopathy, in cultured cells.

    CNM/MTM — Laporte

    MDA has awarded a research grant totaling $341,250 over three years to Jocelyn Laporte at the University of Strasbourg, France. The new funds will help support Laporte’s efforts to identify genes responsible for centronuclear/myotubular myopathies (CNM)/(MTM).

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  • Dongsheng Duan (DMD)

    DMD — Duan

    Dongsheng Duan, professor in the department of molecular microbiology & immunology at the University of Missouri in Columbia, has received an MDA grant totaling $527,670 over three years. The funds will help support Duan's continued research into gene therapy in Duchenne muscular dystrophy DMD).

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  • David Goldhamer (DMD)

    DMD — Goldhamer

    MDA has awarded a research grant totaling $375,000 over three years to David Goldhamer, associate professor, director of the Center for Regenerative Biology, and associate director of the UConn Stem Cell Institute at the University of Connecticut in Storrs. The new funds will help support Goldhamer’s study of muscle stem cells and the repair of damaged muscle in Duchenne muscular dystrophy (DMD).

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  • Bradley Olwin (DMD): Depicted a mouse muscle that was transplanted and examined after two months, and one that was transplanted and examined after two years. The transplanted muscles and the non-transplanted muscles in the other legs are both shown. The muscle size dramatically increases after transplantation and remains large for the lifetime of the mouse.

    DMD — Olwin

    MDA has awarded a research grant totaling $369,165 over three years to Bradley Olwin, professor of molecular, cellular & developmental biology at the University of Colorado in Boulder. The new funds will help support Olwin’s study of muscle regeneration in injured and diseased skeletal muscle — particularly in the muscular dystrophies, including Duchenne muscular dystrophy (DMD).

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  • Grace Pavlath (DMD): Skeletal muscle is composed of myofibers, which are long cylindrical cells. When myofibers are injured, they can degenerate and regenerate from stem cells. However, in many neuromuscular diseases muscle, regeneration doesn't work correctly, and myofibers often adopt an abnormal structure containing numerous cellular "branches."

    DMD — Pavlath

    MDA has awarded a research grant totaling $295,269 over three years to Grace Pavlath, professor in the department of pharmacology at Emory University in Atlanta. The new funds will help support Pavlath’s study of abnormal muscle regeneration in the muscular dystrophies, particularly Duchenne muscular dystrophy (DMD).

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  • Pier Lorenzo Puri (DMD): Muscle cells from a DMD research mouse model; (left) untreated and (right) muscle treated with a histone deacetylase inhibitor called TSA.

    DMD — Puri

    MDA has awarded a research grant totaling $309,336 over three years to Pier Lorenzo Puri, professor of pediatrics at the University of California, San Diego, and associate professor of muscle development and regeneration at the Sanford-Burnham Medical Research Institute, both in La Jolla, Calif. The funds will help support Puri's study of the molecular underpinnings of, and the identification of treatments for, Duchenne muscular dystrophy (DMD).

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  • Hao Shi (DMD)

    DMD — Shi

    MDA has awarded a research development grant totaling $180,000 over three years to Hao Shi, associate research scientist at Yale University in New Haven, Conn. The new funds will help support Shi’s study of muscle repair and regeneration in Duchenne muscular dystrophy (DMD).

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  • Young-Jin Son (DMD)

    DMD — Son

    MDA has awarded a research grant totaling $375,000 over three years to Young-Jin Son, associate professor of developmental neurobiology at Temple University in Philadelphia. The new funds will help support Son’s study of nerve and muscle interaction in muscle diseases such as Duchenne muscular dystrophy (DMD).

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  • Jen-Chywan Wang (DMD)

    DMD — Wang

    MDA has awarded a research grant totaling $220,000 over two years to Jen-Chywan Wang, assistant professor at the University of California, Berkeley. The new funds will help support Wang’s study of the effects of chronic glucocorticoid (steroid) treatment in Duchenne muscular dystrophy (DMD).

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  • Xander Wehrens (DMD)

    DMD — Wehrens

    MDA has awarded a research grant totaling $313,500 to Xander Wehrens, associate professor in the departments of molecular physiology & biophysics and medicine at Baylor College of Medicine in Houston, Texas. The new funds will help support Wehrens’ research into abnormal heart function in Duchenne muscular dystrophy (DMD).

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  • Harol Bernstein (DMD/BMD): (Left) A section of mouse leg muscle shows normal muscle fibers, with muscle cell nuclei shown in blue and aligned properly at the edge of muscle fibers. (Right) A similar mouse leg muscle in which human embryonic stem cells, distinguished by green fluorescence and centralized nuclei, have been injected.

    DMD/BMD — Bernstein

    Harold Bernstein, professor of pediatrics at the University of California, San Francisco, has been awarded an MDA research grant totaling $540,000 over three years. The award will help support Bernstein's study of human muscle development and potential cell-based therapies for treatment of degenerative muscle diseases such as Duchenne muscular dystrophy (DMD).

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  • Andrew Brack (DMD/BMD)

    DMD/BMD — Brack

    Andrew Brack, an assistant professor at the Center for Regenerative Medicine at Massachusetts General Hospital in Boston, was awarded an MDA research grant totaling $353,259 over three years. The funds will help support Brack's research into the function of adult muscle stem cells called "satellite cells" in Duchenne muscular dystrophy (DMD) and possibly in other muscular dystrophies as well.

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  • Masahiko Hoshijima (DMD/BMD): An imaging technique called high-resolution Electron Microscope (EM) Tomography provides a 3D rendering of structural heart-muscle cell abnormalities characteristic in BIO14.6 hamsters.

    DMD/BMD — Hoshijima

    MDA awarded a grant totaling $367,386 over three years to Masahiko Hoshijima, professor of medicine at the University of California, San Diego in La Jolla, Calif. The funds will help support Hoshijima's research into connections between cardiac and respiratory failure in muscle diseases such as Duchenne (DMD), Becker (BMD) and other muscular dystrophies (MDs).

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  • David Kass (DMD/BMD)

    DMD/BMD — Kass

    MDA has awarded a research grant totaling $210,618 over two years to David Kass, professor of cardiology, medicine and biomedical engineering at Johns Hopkins University in Baltimore. The new funds will help support Kass’ study of the disease process and potential therapeutic strategies in Duchenne (DMD), Becker (BMD) and other muscular dystrophies.

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  • Jianming Liu (DMD/BMD): A cross-section of skeletal muscle from a research mouse engineered to express high levels of alpha-7 integrin. The distribution of the transgenic genes' products suggests only a limited amount of its binding partner beta-1 integrin in the skeletal muscle, and identified a bottle-neck factor in the development of integrin-based therapy for muscular dystrophy.

    DMD/BMD — Liu

    MDA has awarded a research development grant totaling $179,912 over three years to Jianming Liu in the department of cell and tissue biology at the University of California, San Francisco. The new funds will help support Liu’s study of the disease process in Duchenne (DMD) and  Becker (BMD) muscular dystrophies. Using research mouse models for DMD and BMD, Liu and colleagues plan to investigate a potential stem-cell-based therapy for the two diseases using engineered stem cells designed to help regenerate healthy skeletal muscle tissues.

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  • Giovanni Coppola (FA)

    FA — Coppola

    MDA has awarded a research grant totaling $398,541 to Giovanni Coppola, assistant professor and co-director at the University of California, Los Angeles, Informatics Center for Neurogenetics and Neurogenomics. The funds will help support Coppola's research into biological indicators, called "biomarkers," in Friedreich's ataxia (FA).

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  • Joseph Sarsero (FA)

    FA — Sarsero

    MDA has awarded a research grant totaling $416,250 over three years to Joseph Sarsero, head of the Friedreich Ataxia Laboratory Research Program at the Murdoch Childrens Research Institute in Parkville, Victoria, Australia. The new funds will help support Sarsero’s development of a new mouse model of Friedreich’s ataxia (FA).

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  • Steven Greenberg (IBM): Mislocalization of TDP43 protein causes degeneration in the eyes of 1-day-old fruit flies. Greenberg, and co-principal investigator J. Paul Taylor, will examine the effects of similar TDP43 redistribution in inclusion-body myositis, with a particular focus on whether it causes damage to muscle fibers.

    IBM — Greenberg

    MDA has awarded a grant totaling $444,314 over a period of three years to Steven Greenberg at Brigham and Women's Hospital and Harvard Medical School in Boston. The funds will help support Greenberg's study of the role of a protein called TDP43 in sporadic and hereditary inclusion-body myositis (IBM).

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  • William Atchison (LEMS)

    LEMS — Atchison

    MDA awarded a research grant totaling $336,503 to William Atchison, professor of pharmacology/toxicology and acting dean for research at Michigan State University College of Veterinary Medicine in East Lansing. The funds will support Atchison's research into the biological mechanisms underlying Lambert-Eaton myasthenic syndrome (LEMS).

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  • Michael Hauser (LGMD): Pictured Zebrafish model injected with (left) nothing, (middle) human myotilin gene (MYOT) and (right) the myotilin gene containing the T571 mutation that causes LGMD1A. (Photo credit: Erica Davis, Ph.D.)

    LGMD — Hauser

    Michael Hauser, an associate professor in the Section of Medical Genetics, Department of Medicine at Duke University Medical Center in Durham, N.C., has been awarded an MDA grant totaling $383,856 over a period of three years. The funds will help support Hauser's efforts to identify mutations responsible for dominantly inherited (type 1) limb-girdle muscular dystrophy (LGMD). (Dominant inheritance means only one gene mutation, inherited from one parent, is sufficient to cause the disease. As a result, offspring of affected individuals have a 50 percent probability of inheriting the condition from their parents.)

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  • Peter Kang (LGMD)

    LGMD — Kang

    MDA has awarded a research grant totaling $299,722 over three years to Peter Kang, assistant professor of neurology at Harvard Medical School and director of the electromyography laboratory at Children’s Hospital Boston. The funds will help support Kang’s research into identification of gene mutations that can cause limb-girdle muscular dystrophy (LGMD).

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  • Mark Rich (MC)

    MC — Rich

    MDA has awarded a research grant totaling $53,358 to Mark Rich, an associate professor at Wright State University in Dayton, Ohio. The new funds will help support Rich’s study of the disease process in myotonia congenita (MC).

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  • Xiaohua Wu (MD): A snapshot of the genesis of muscle cells in the laboratory. Immature muscle cells called myoblasts (red) fuse together to become myotubes with multiple nuclei (blue). Laminins, which play an important role in cell maturation and migration and survival, are stained green.

    MD — Wu

    MDA has awarded a research grant totaling $224,863 over three years to Xiaohua Wu, research faculty for the drug discovery group at the McColl-Lockwood Laboratory for Muscular Dystrophy Research at Carolinas Medical Center in Charlotte, N.C. The new funds will help support Wu’s research into effective therapies for various types of muscular dystrophy (MD).

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  • Chi Wai Lee (MG)

    MG — Lee

    MDA has awarded a research development grant totaling $180,000 over three years to Chi Wai Lee, postdoctoral fellow in the department of cell biology at Emory University School of Medicine in Atlanta. The grant will help support Lee’s research into therapeutic strategies to ameliorate the symptoms in myasthenia gravis (MG).

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  • Jon Lindstrom (MG)

    MG — Lindstrom

    MDA has awarded a research grant totaling $450,000 over three years to Jon Lindstrom, professor of neuroscience and pharmacology at the Medical School of the University of Pennsylvania in Philadelphia. The new funds will help support Lindstrom’s continued efforts to develop an immunosuppresive therapy for myasthenia gravis (MG).

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  • Pragna Patel (MG)

    MG — Patel

    MDA has awarded a research grant totaling $121,139 to Pragna Patel, professor at the Institute for Genetic Medicine, Keck School of Medicine, at the University of Southern California in Los Angeles. The new funds will help support Patel’s research aimed at determining the faulty gene responsible for myasthenia gravis (MG) in a particular family with the disease.

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  • John Lueck (MMD)

    MMD — Lueck

    MDA has awarded a research development grant totaling $180,000 over three years to John Lueck, a postdoctoral fellow at the University of Iowa Carver College of Medicine in Iowa City. The new funds will help support Lueck’s research into the mechanisms responsible for muscle weakness and degeneration in type 1 myotonic muscular dystrophy (MMD1, or DM1).

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  • Mani Mahadevan (MMD)

    MMD — Mahadevan

    MDA has awarded a research grant totaling $435,000 over three years to Mani Mahadevan, professor in the department of pathology, medical director of the molecular diagnostics lab and associate director of the cytogenics lab at the University of Virginia in Charlottesville. The new funds will help support Mahadevan’s study of the underlying molecular mechanisms in type 1 myotonic muscular dystrophy (MMD1, or DM1).

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  • Araya Puwanant (MMD)

    MMD — Puwanant

    MDA has awarded a clinical research training grant totaling $173,400 to Araya Puwanant at the University of Rochester (New York) Medical Center. The new funds will support completion of a two-year fellowship during which Puwanant will study the disease process in myotonic muscular dystrophy (MMD, or DM).

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  • James Dowling (MTM): Pictured (top left) Dowling in his office; (top right) a muscle biopsy sample taken from a person with MTM — the large central nuclei are the defining biological features of the disease. Both bottom photos depict muscle stained with alpha bungarotoxin, a marker for the neuromuscular junction. On the left is muscle from a mouse unaffected by MTM. To the right is muscle from an MTM-affected mouse, in which the junctions clearly are significantly larger.

    MTM — Dowling

    MDA has awarded a research grant totaling $353,679 to James Dowling, assistant professor in the departments of pediatrics and neurology at the University of Michigan Medical Center in Ann Arbor. The funds will help support Dowling's research into potential therapies for myotubular myopathy (MTM).

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  • Antoni Barrientos (Mito. Myopothy)

    Mito. Myopathy — Barrientos

    MDA has awarded a research grant totaling $346,500 over three years to Antoni Barrientos, an associate professor in the departments of neurology and biochemistry and molecular biology at the University of Miami (Florida) Miller School of Medicine. The grant will help support Barrientos' study of the underlying molecular mechanisms in some forms of mitochondrial myopathy.

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  • J. Paul Taylor (SBMA)

    SBMA — Taylor

    J. Paul Taylor, associate member of St. Jude Children’s Research Hospital in Memphis, Tenn., has received an MDA research grant totaling $330,000 over three years. The funds will help support Taylor’s continued research into a number of possible therapeutic targets in spinal-bulbar muscular atrophy (SBMA).

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  • Claudio Sette (SMA): Pictured neural (nervous system) stem cells grown in conditions that encourage them to mature into motor neurons. The cells, once isolated and grown, were analyzed for neuronal markers (green) and photographed using a confocal microscope. The blue staining (upper left) shows cell nuclei; the green staining marks neurons; and the red staining shows that the neuron has migratory potential. Bottom right: A merged image of the three other panels indicates the strong potential of neural ste

    SMA — Sette

    MDA has awarded a research grant totaling $219,000 over three years to Claudio Sette, associate professor for the department of public health and cell biology at the University of Rome Tor Vergata in Rome, Italy. The new funds will help support Sette’s study of the molecular mechanisms underlying spinal muscular atrophy (SMA).

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In December 2010, the MDA Board of Directors funded 44 research projects targeting nearly two dozen neuromuscular diseases. These projects are in addition to some 330 grants currently funded by MDA.

For an overview of grants awarded by MDA in Winter 2011, see:

  • MDA Awards $13.5 Million in Research Grants
  • Eight New Grants Bolster MDA's Battle Against ALS

For a list of the more than 330 grants currently being funded by MDA, view this PDF.

ALS — Barrett ›

Grants at a Glance

Summer 2012
Winter 2012
Summer 2011
Winter 2011
Summer 2010

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    • Volume 1, Issue 1, October 2011
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Grants at a Glance — Winter 2011

  • ALS — Barrett
  • ALS — Berry
  • ALS — Berthod
  • ALS — Israelson
  • ALS — Muotri
  • ALS — Sockanathan
  • ALS — Wang
  • ALS/SMA — Miller
  • CMD — Durbeej-Hjalt
  • CMD — Lamande
  • CMT — Nguyen
  • CNM/MTM — Laporte
  • DMD — Duan
  • DMD — Goldhamer
  • DMD — Olwin
  • DMD — Pavlath
  • DMD — Puri
  • DMD — Shi
  • DMD — Son
  • DMD — Wang
  • DMD — Wehrens
  • DMD/BMD — Bernstein
  • DMD/BMD — Brack
  • DMD/BMD — Hoshijima
  • DMD/BMD — Kass
  • DMD/BMD — Liu
  • FA — Coppola
  • FA — Sarsero
  • IBM — Greenberg
  • LEMS — Atchison
  • LGMD — Hauser
  • LGMD — Kang
  • MC — Rich
  • MD — Wu
  • MG — Lee
  • MG — Lindstrom
  • MG — Patel
  • MMD — Lueck
  • MMD — Mahadevan
  • MMD — Puwanant
  • MTM — Dowling
  • Mito. Myopathy — Barrientos
  • SBMA — Taylor
  • SMA — Sette
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