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Grants at a Glance — Summer 2012

  • Alex Parker (ALS): Green fluorescent protein illuminates the muscle-controlling nerve cells called motor neurons in a C. elegans (nematode, or roundworm) research model of amyotrophic lateral sclerosis.

    ALS — Alex Parker, Ph.D.

    MDA awarded a research grant totaling $231,300 over three years to Alex Parker, assistant professor in the department of pathology and cellular biology at the University of Montreal Hospital Research Center in Montreal, Quebec (Canada). The funds will help support Parker's study of cellular stress response in neurodegeneration associated with amyotrophic lateral sclerosis (ALS).

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  • Christine Vande Velde (ALS): Cell showing stress granules; TDP43 is green; stress granule markers G3BP are red; TIA-1 is blue; overlap of all three is purple.

    ALS — Christine Vande Velde, Ph.D.

    MDA awarded a research grant totaling $358,242 over three years to Christine Vande Velde, research assistant professor in the department of medicine at the University of Montreal Hospital Research Center in Montreal, Quebec (Canada). The funds will help support Vande Velde’s study of the role of TDP43 and the stress granule mechanism in amyotrophic lateral sclerosis (ALS).

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  • Marc Weisskopf (ALS)

    ALS — Marc Weisskopf, Sc.D., Ph.D.

    MDA awarded a research grant totaling $301,614 over three years to Marc Weisskopf, associate professor of environmental and occupational epidemiology in the departments of environmental health and epidemiology at Harvard School of Public Health in Boston. The funds will help support Weisskopf’s search for nongenetic risk factors for amyotrophic lateral sclerosis (ALS).

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  • Dwight Koeberl (AMD) with colleagues; Koeberl is on far right.

    AMD — Dwight Koeberl, M.D., Ph.D.

    MDA awarded a research grant totaling $253,812 over two years to Dwight Koeberl, associate professor in the department of pediatrics at Duke University Medical Center in Durham, N.C. The funds will help support Koeberl’s research on the development of gene therapy for acid maltase deficiency (AMD, or Pompe disease).

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  • Kevin Campbell (CMD)

    CMD — Kevin Campbell, Ph.D.

    MDA awarded a research grant totaling $375,000 over three years to Kevin Campbell, professor of neurology and internal medicine at the University of Iowa in Iowa City. The funds will help support Campbell's study of a process called protein O-mannosylation in a mouse model of congenital muscular dystrophy (CMD).

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  • Madhuri Hegde (CMD)

    CMD — Madhuri Hegde, Ph.D.

    Madhuri Hegde, associate professor and scientific director at Emory Genetics Laboratory, Emory University in Atlanta, was awarded an MDA research grant totaling $262,928 over two years to identify genes that, when mutated, cause congenital muscular dystrophy (CMD).

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  • Paul Brehm (CMS) with Dr. Mike Linhoff

    CMS — Paul Brehm, Ph.D.

    Paul Brehm, senior scientist at Oregon Health Science University in Portland, was awarded an MDA research grant totaling $351,648 over three years to study the underlying mechanisms of movement disorders in some forms of congenital myasthenic syndromes (CMS).

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  • Bogdan Beirowski (CMT): Pictured behind him is a painting depicting an artistic interpretation of a degenerating axon. Inset: A view of degenerating axons from a mouse model of CMT via fluorescence (top) and electron microscopy (bottom).

    CMT — Bogdan Beirowski, M.D., Ph.D.

    Postdoctoral research scholar Bogdan Beirowski, in the department of genetics at the Washington University School of Medicine in St. Louis, was awarded an MDA development grant totaling $180,000 over three years to study how defective Schwann cells lead to nerve-cell damage in Charcot-Marie-Tooth disease (CMT).

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  • Stephan Züchner (CMT)

    CMT — Stephan Züchner, M.D.

    Stephen Züchner, associate professor of human genetics and neurology at the University of Miami Miller School of Medicine in Florida, was awarded an MDA research grant totaling $390,000 over three years to identify genes responsible for Charcot-Marie-Tooth disease (CMT).

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  • Jeffrey Milbrandt (CMT/FA)

    CMT/FA — Jeffrey Milbrandt, M.D., Ph.D.

    MDA awarded a research grant totaling $357,366 over three years to Jeffrey Milbrandt, professor and head of the department of genetics, and professor of pathology & immunology, medicine and neurology at Washington University School of Medicine in St. Louis.

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  • Tathagata Chaudhuri (DMD): Adult human stem cells pictured have been "grown" for five days and are on the path to becoming muscle cells.

    DMD — Tathagata Chaudhuri, Ph.D.

    Postdoctoral research fellow Tathagata Chaudhuri, at the Perelman School of Medicine at the University of Pennsylvania in Philadelphia, was awarded an MDA development grant (DG) totaling $180,000 over three years to develop a stem cell therapy for muscular dystrophies, including Duchenne muscular dystrophy (DMD), that will lead to muscle regeneration.

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  • Adam Engler (DMD/BMD): This high-resolution image, taken using a microscopy technique called immunofluorescence, shows adipose-derived stem cells that have fused together to become muscle. The microtubule network is green; cell nuclei are blue.

    DMD/BMD — Adam Engler, Ph.D.

    Adam Engler, assistant professor at the University of California, San Diego, in La Jolla, was awarded an MDA research grant totaling $390,000 over three years to study cell-based therapies designed for Duchenne (DMD) and Becker (BMD) muscular dystrophies and other muscle diseases.

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  • Atsushi Asakura (DMD/BMD): Asakura uses an inverted microscope to capture images of satellite cells, which play essential roles in muscle regeneration.

    DMD/BMD — Atsushi Asakura, Ph.D.

    Atsushi Asakura, assistant professor at the University of Minnesota Medical School in Minneapolis, was awarded an MDA research grant totaling $378,531 over three years to study how the system of blood vessels known as the vasculature affects muscles in Duchenne (DMD) and Becker (BMD) muscular dystrophies.

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  • Daniel Michele (DMD/BMD)

    DMD/BMD — Daniel Michele, Ph.D.

    Daniel Michele, associate professor in the department of molecular and integrative physiology at the University of Michigan in Ann Arbor, was awarded an MDA research grant totaling $364,965 over three years to study the underlying mechanisms of muscle weakness and muscle fatigue in Duchenne (DMD) and Becker (BMD) muscular dystrophies. Michele's work could have implications for other dystrophies as well, including limb-girdle muscular dystrophy (LGMD) and congenital muscular dystrophy (CMD).

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  • David Gokhin (DMD/BMD): In this image, taken with a microscopy technique called transmission electron microscopy, the "stair-step" pattern indicates unusually high misalignment of myofibrils (the working parts of muscle fibers) in a leg muscle from a mouse that has no gamma-actin protein.

    DMD/BMD — David Gokhin, Ph.D.

    Research associate David Gokhin at the Scripps Research Institute in La Jolla, Calif., was awarded an MDA development grant totaling $180,000 over three years to study the role of a protein called gamma-actin in muscle degeneration and weakness in Duchenne (DMD) and Becker (BMD) muscular dystrophies.

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  • Dean Burkin (DMD/BMD)

    DMD/BMD — Dean Burkin, Ph.D.

    Dean Burkin, associate professor of pharmacology at the University of Nevada School of Medicine in Reno, was awarded an MDA research grant totaling $308,028 over three years to study laminin-111 protein therapy for Duchenne (DMD) and Becker (BMD) muscular dystrophies.

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  • Joseph Metzger (DMD/BMD): illustration of a molecular "Band-Aid"

    DMD/BMD — Joseph Metzger, Ph.D.

    MDA awarded a research grant totaling $237,868 over two years to Joseph Metzger, professor and chair of integrative biology and physiology at the University of Minnesota in Minneapolis. The funds will help support Metzger's work in development and testing of copolymer molecules, or "membrane sealants," for Duchenne (DMD), Becker (BMD) and other muscular dystrophies.

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  • Rita Perlingeiro (DMD/BMD) and colleague

    DMD/BMD — Rita Perlingeiro, Ph.D.

    MDA awarded a research grant totaling $390,000 over three years to Rita Perlingeiro, associate professor in the Department of Medicine at the University of Minnesota in Minneapolis. The funds will help support Perlingeiro's study of genetic correction and muscle regeneration in Duchenne (DMD) and Becker (BMD) muscular dystrophies.

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  • Ryan Wuebbles (DMD/BMD)

    DMD/BMD — Ryan Wuebbles, Ph.D.

    Ryan Wuebbles, a postdoctoral fellow in pharmacology at the University of Nevada School of Medicine in Reno, was awarded an MDA development grant (DG) totaling $180,000 over three years to study the potential use of a protein called laminin-111 as the basis of therapies for Duchenne (DMD) and Becker (BMD) muscular dystrophies.

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  • Tom Thompson (DMD/BMD): The myostatin protein inhibits the size and mass of muscle. Thompson is studying several strategies for myostatin inhibition, including use of a protein called follistatin that facilitates myostatin degradation.

    DMD/BMD — Tom Thompson, Ph.D.

    Tom Thompson, associate professor in the department of molecular genetics, biochemistry and microbiology at the University of Cincinnati in Ohio, was awarded an MDA research grant totaling $330,000 over three years. The funds will help support Thompson's study of potential therapies based on blocking a protein called myostatin for Duchenne (DMD) and Becker (BMD) muscular dystrophies.

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  • Veronica Hinton (DMD/BMD)

    DMD/BMD — Veronica Hinton, Ph.D.

    Veronica Hinton, associate professor of clinical neuropsychology at Columbia University in New York, was awarded an MDA research grant totaling $397,596 over three years to study cognitive problems in children with dystrophinopathies, which include Duchenne (DMD) and Becker (BMD) muscular dystrophies.

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  • Mary Baylies (EDMD): Left panel: Thirty distinct muscle fibers are formed in each abdominal segment. Middle panel: The 30 muscle fibers from one segment are shown under high magnification. Right panel: A schematic shows the 30 distinct fibers that make up one segment.

    EDMD — Mary Baylies, Ph.D.

    Mary Baylies, professor in the program of developmental biology at Sloan-Kettering Institute, Memorial Sloan-Kettering Cancer Center in New York, was awarded an MDA research grant totaling $399,269 over three years to study LMNA gene mutations and the role of a protein called esconsin in Emery-Dreifuss muscular dystrophy (EDMD).

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  • Yosef Gruenbaum (EDMD/LGMD/CMT)

    EDMD/LGMD/CMT — Yosef Gruenbaum, Ph.D.

    MDA awarded a research grant totaling $300,009 over three years to Yosef Gruenbaum, professor and elected chairman at the Alexander Silberman Institute of Life Sciences, Hebrew University of Jerusalem, in Israel. The funds will help support Gruenbaum’s study of proteins called lamins and their role in muscle diseases such as Emery-Dreifuss (EDMD) and limb-girdle (LGMD) muscular dystrophies, and Charcot-Marie-Tooth disease (CMT).

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  • Joel Chamberlain (FSHD)

    FSHD — Joel Chamberlain, Ph.D.

    Joel Chamberlain, research assistant professor in the department of medicine at the University of Washington in Seattle, was awarded an MDA research grant totaling $330,780 over three years to study a therapeutic approach called RNA interference (RNAi) for the treatment of facioscapulohumeral muscular dystrophy (FSHD).

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  • Rossella Tupler (FSHD)

    FSHD — Rossella Tupler, M.D., Ph.D.

    MDA awarded a research grant totaling $260,000 over two years to Rossella Tupler, research assistant professor in the program of molecular medicine at the University of Massachusetts Medical School in Worcester. The funds will help support Tupler’s search for the molecular cause of facioscapulohumeral muscular dystrophy (FSHD).

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  • Melissa Spencer (LGMD): A depiction of the biomolecular structure of the calpain 3 protein that shows the molecule's four different "domains." The regions shown in red and purple are unique to calpain 3 and are not observed in other proteins, but mutations in any of the four domains can lead to LGMD2A.

    LGMD — Melissa Spencer, Ph.D.

    Melissa Spencer, professor of neurology at the David Geffen School of Medicine at University of California, Los Angeles, was awarded an MDA research grant totaling $390,000 over three years to study the role of an enzyme called calpain 3 in type 2A limb-girdle muscular dystrophy (LGMD2A).

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  • Feng Lin (MG)

    MG — Feng Lin, Ph.D.

    Feng Lin, associate professor in the department of pathology at Case Western Reserve University School of Medicine in Cleveland, Ohio, was awarded an MDA research grant totaling $390,000 over three years to study a potential new cell-based therapy for myasthenia gravis (MG).

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  • JianRong Sheng (MG): Sheng prepares the media used to culture immune cells, such as T cells and B cells, taken from mice with a disease resembling myasthenia gravis.

    MG — JianRong Sheng, Ph.D.

    JianRong Sheng, assistant professor in the department of neurology and rehabilitation at the University of Illinois at Chicago Medical Center, was awarded an MDA research grant totaling $317,058 over three years. The funds will help support Sheng's study of potential therapeutic treatments for myasthenia gravis (MG).

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  • Lin Mei (MG)

    MG — Lin Mei, M.D., Ph.D.

    MDA awarded a research grant totaling $390,000 over three years to Lin Mei, professor and director of the Institute of Molecular Medicine and Genetics at Georgia Health Sciences University in Augusta. The funds will help support Mei’s research on the role of a protein called LRP4 in myasthenia gravis (MG).

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  • Charles Thornton (MMD)

    MMD — Charles Thornton, M.D.

    Charles Thornton, professor of neurology at the University of Rochester in New York, was awarded an MDA research grant totaling $308,935 over three years. The funds will help support Thornton's work to expedite the development of effective treatments for type 1 myotonic muscular dystrophy (MMD, also known as DM).

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  • Thurman Wheeler (MMD): A fluorescence microscope image showing advanced muscle disease in a mouse model of myotonic dystrophy. Muscle fibers are outlined in green and muscle nuclei are highlighted in blue.

    MMD — Thurman Wheeler, M.D.

    Thurman Wheeler, assistant professor in the department of neurology and Center for Neural Development & Disease at the University of Rochester, New York, was awarded an MDA research grant totaling $396,000 over three years to identify potential therapies aimed either at slowing down the progression of muscle degeneration or improving muscle health in type 1 myotonic muscular dystrophy (MMD1, or DM1).

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  • Michio Hirano (Mito. Myopathy) with Caterina Garone

    Mito. Myopathy — Michio Hirano, M.D.

    MDA awarded a research grant totaling $398,532 over three years to Michio Hirano, professor of neurology and chief of the neuromuscular division at Columbia University Medical Center in New York. The funds will help support Hirano’s study of “molecular bypass therapy” for a mitochondrial myopathy called thymidine kinase 2 (TK2) deficiency.

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  • Andrew Lieberman (SBMA)

    SBMA — Andrew Lieberman, M.D., Ph.D.

    Andrew Lieberman, assistant professor of pathology at the University of Michigan Medical School in Ann Arbor, was awarded an MDA research grant totaling $405,000 over three years to study a new therapy approach for spinal-bulbar muscular atrophy (SBMA).

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On Aug. 1, 2012, the MDA Board of Directors approved funding for 33 research projects targeting more than 18 neuromuscular diseases, as well as general muscle disease research with implications for many of the diseases in MDA's program. The newly funded projects are part of some 300 research projects currently supported by MDA.

For an overview of grants awarded by MDA in August 2012, see:

  • MDA Commits $10.7 Million to Neuromuscular Disease Research
  • ALS: New MDA Grants Focus on Multisystem Aspects of the Disease

For a complete list of current MDA-funded research projects, view this PDF.

ALS — Alex Parker, Ph.D. ›

Grants at a Glance

Summer 2012
Winter 2012
Summer 2011
Winter 2011
Summer 2010

Research

  • Research News
  • Active Research Grants
  • Research Grants Programs
  • MDA Translational Research Program
    • Clinical Research Training Grant (CRTG)
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  • Opportunities for Researchers
  • Annual Conference Series
  • Grants at a Glance — Winter 2013
  • MDA Research Contact
  • Helpful Links
  • Clinical Trials and Studies
  • Clinical Research Network Grant
  • Newborn Screening for Neuromuscular Diseases
  • MyoBlast Research Newsletter
    • Volume 1, Issue 1, October 2011
    • Volume 2, Issue 1, February 2012

Grants at a Glance — Summer 2012

  • ALS — Alex Parker, Ph.D.
  • ALS — Christine Vande Velde, Ph.D.
  • ALS — Marc Weisskopf, Sc.D., Ph.D.
  • AMD — Dwight Koeberl, M.D., Ph.D.
  • CMD — Kevin Campbell, Ph.D.
  • CMD — Madhuri Hegde, Ph.D.
  • CMS — Paul Brehm, Ph.D.
  • CMT — Bogdan Beirowski, M.D., Ph.D.
  • CMT — Stephan Züchner, M.D.
  • CMT/FA — Jeffrey Milbrandt, M.D., Ph.D.
  • DMD — Tathagata Chaudhuri, Ph.D.
  • DMD/BMD — Adam Engler, Ph.D.
  • DMD/BMD — Atsushi Asakura, Ph.D.
  • DMD/BMD — Daniel Michele, Ph.D.
  • DMD/BMD — David Gokhin, Ph.D.
  • DMD/BMD — Dean Burkin, Ph.D.
  • DMD/BMD — Joseph Metzger, Ph.D.
  • DMD/BMD — Rita Perlingeiro, Ph.D.
  • DMD/BMD — Ryan Wuebbles, Ph.D.
  • DMD/BMD — Tom Thompson, Ph.D.
  • DMD/BMD — Veronica Hinton, Ph.D.
  • EDMD — Mary Baylies, Ph.D.
  • EDMD/LGMD/CMT — Yosef Gruenbaum, Ph.D.
  • FSHD — Joel Chamberlain, Ph.D.
  • FSHD — Rossella Tupler, M.D., Ph.D.
  • LGMD — Melissa Spencer, Ph.D.
  • MG — Feng Lin, Ph.D.
  • MG — JianRong Sheng, Ph.D.
  • MG — Lin Mei, M.D., Ph.D.
  • MMD — Charles Thornton, M.D.
  • MMD — Thurman Wheeler, M.D.
  • Mito. Myopathy — Michio Hirano, M.D.
  • SBMA — Andrew Lieberman, M.D., Ph.D.
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