
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 2016
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CMD - Douglas Anderson, Ph.D.
Grant Announced: Winter 2016Disease Type: Congenital Muscular Dystrophy (CMD)““The continued discovery and characterization of key regulators of skeletal muscle function, such as myoregulin, will ...”
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ALS - Paul August, Ph.D.
Grant Announced: Winter 2016Disease Type: Amyotrophic Lateral Sclerosis (ALS)“Paul August is using novel technology to develop a chip that will facilitate the testing of experimental therapies for A...”
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MM - Antoni Barrientos, Ph.D.
Grant Announced: Winter 2016Disease Type: Mitochondrial Myopathies (MM)““The study of mitochondria is now a very ‘hot issue’ for both basic and clinical research, Antoni Barrientos says....”
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ALS - Asim Beg, Ph.D.
Grant Announced: Winter 2016Disease Type: Amyotrophic Lateral Sclerosis (ALS)““The devastating impact that ALS has on patients and their families is a major factor that has inspired me to study th...”
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nemaline myopathy - Alan Beggs, Ph.D.
Grant Announced: Winter 2016Disease Type: Endocrine Myopathies“Alan Beggs is working to uncover genes that cause nemaline myopathy.”
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Periodic Paralysis - Steve Cannon, M.D., Ph.D.
Grant Announced: Winter 2016Disease Type: Endocrine Myopathies“Steve Cannon is investigating the mechanistic basis for exercise-induced weakness in periodic paralysis.”
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MMD - Matthew Disney, Ph.D.
Grant Announced: Winter 2016Disease Type: Myotonic Dystrophy (DM)““We are getting closer by the day to a treatment,” Matthew Disney says. “I know it is a long process, but patients...”
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CCD - James Dowling, M.D., Ph.D.
Grant Announced: Winter 2016Disease Type: Endocrine Myopathies“James Dowling is working on a drug development platform that will help identify drugs suitable for patients with CCD and...”
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MM - Pradeep Reddy Dubbaka Venu, Ph.D.
Grant Announced: Winter 2016Disease Type: Mitochondrial Myopathies (MM)““My interest in mitochondrial diseases began after learning that there are no effective methods available to prevent t...”
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CMD - Mahasweta Girgenrath, Ph.D.
Grant Announced: Winter 2016Disease Type: Congenital Muscular Dystrophy (CMD)“Mahasweta Girgenrath is working to determine the potential of a dual treatment in laminin-deficient congenital muscular...”
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CCD - Susan Hamilton, Ph.D.
Grant Announced: Winter 2016Disease Type: Endocrine Myopathies““Through my many interactions with MDA, I have talked to families of patients with CCD and learned about the consequen...”
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ALS - Claudio Hetz, Ph.D.
Grant Announced: Winter 2016Disease Type: Amyotrophic Lateral Sclerosis (ALS)““Patients afflicted with ALS now need to know that we are working for them, even though our current scientific efforts...”
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CMT - Henry Houlden, M.D., Ph.D.
Grant Announced: Winter 2016Disease Type: Charcot-Marie-Tooth Disease (CMT)““My focus is on finding genes and disease pathways,” Henry Houlden says. “By identifying genes we can give patient...”
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FSHD - Peter Jones, Ph.D.
Grant Announced: Winter 2016Disease Type: Facioscapulohumeral Muscular Dystrophy (FSH, FSHD)“Peter Jones, and his wife and co-principal investigator Takako Jones, are focused on therapeutic development and preclin...”
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ALS - Nam Chul Kim, Ph.D.
Grant Announced: Winter 2016Disease Type: Amyotrophic Lateral Sclerosis (ALS)“Nam Chul Kim’s work could have impact in a number of diseases including amyotrophic lateral sclerosis (ALS), frontotem...”
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ALS - Evangelos Kiskinis, Ph.D.
Grant Announced: Winter 2016Disease Type: Amyotrophic Lateral Sclerosis (ALS)““The main focus of my lab is to elucidate the molecular pathways that lead to the degeneration of the spinal motor neu...”
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FSHD - Louis Kunkel, Ph.D.
Grant Announced: Winter 2016Disease Type: Facioscapulohumeral Muscular Dystrophy (FSH, FSHD)“Louis Kunkel is working to identify genetic modifiers of facioscapulohumeral muscular dystrophy.”
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DMD - Gabsang Lee, D.V.M, Ph.D.
Grant Announced: Winter 2016Disease Type: Duchenne Muscular Dystrophy (DMD)“Although several animal models are available for DMD research, disease progression in the animals is somewhat different ...”
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LGMD - Monkol Lek, Ph.D.
Grant Announced: Winter 2016Disease Type: Limb-Girdle Muscular Dystrophy (LGMD)““We now live in a golden period for genetic disease diagnosis where advances in DNA sequencing technology have dramati...”
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SMA - Chris Lorson, Ph.D.
Grant Announced: Winter 2016Disease Type: Spinal Muscular Atrophy (SMA)““SMA is exceptionally well suited for therapeutic approaches that target SMN2; however, there currently is no FDA-appr...”
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DMD - Ke Ma, M.D., Ph.D.
Grant Announced: Winter 2016Disease Type: Duchenne Muscular Dystrophy (DMD)““With the discovery of basic disease mechanisms and new molecules targeting disease pathways involved in disparate asp...”
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ALS - Jim Manley, Ph.D.
Grant Announced: Winter 2016Disease Type: Amyotrophic Lateral Sclerosis (ALS)“Jim Manley and colleagues have found that the absence of senataxin protein leads to a defect in the clearance of unwante...”
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SMA - Steven Markus, Ph.D.
Grant Announced: Winter 2016Disease Type: Spinal Muscular Atrophy (SMA)““The tools we have at our disposal to answer complex biological questions are becoming extremely sophisticated, and en...”
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ALS - Kathrin Meyer, Ph.D.
Grant Announced: Winter 2016Disease Type: Amyotrophic Lateral Sclerosis (ALS)““ALS is very devastating for the patients and their families, and new therapeutic strategies are urgently needed,” K...”
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ALS - Michael Miller, Ph.D.
Grant Announced: Winter 2016Disease Type: Amyotrophic Lateral Sclerosis (ALS)““Technological advances are breaking down barriers to understanding diseases such as ALS,” Michael Miller says. “S...”
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DMD - Bradley Olwin, Ph.D.
Grant Announced: Winter 2016Disease Type: Duchenne Muscular Dystrophy (DMD)“Bradley Olwin is working to determine whether activation of satellite cells in muscle will promote muscle repair in a mo...”
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SMA - Franco Pagani, M.D.
Grant Announced: Winter 2016Disease Type: Spinal Muscular Atrophy (SMA)““Using a novel strategy based on small RNA, we intend to correct the splicing defect of the SMN2 gene,” Franco Pagan...”
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ALS - Alex Parker, Ph.D.
Grant Announced: Winter 2016Disease Type: Amyotrophic Lateral Sclerosis (ALS)““Using simple animal models, we focus on finding genetic pathways contributing to motor neuron degeneration,” Alex P...”
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SMA - Livio Pellizzoni, M.D.
Grant Announced: Winter 2016Disease Type: Spinal Muscular Atrophy (SMA)“Livio Pellizzoni is working to elucidate a new function of SMN protein, which is deficient in spinal muscular atrophy.”
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ALS - Thomas Philips, Ph.D.
Grant Announced: Winter 2016Disease Type: Amyotrophic Lateral Sclerosis (ALS)““Under stress, motor neurons become increasingly vulnerable and dependent on metabolic support by its neighboring glia...”
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SMA - Natalia Rodriguez Muela, Ph.D.
Grant Announced: Winter 2016Disease Type: Spinal Muscular Atrophy (SMA)“Natalia Rodriguez Muela will examine how SMN protein levels are affected by autophagy, a cellular garbage disposal syste...”
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DMD - Michael Rudnicki, Ph.D.
Grant Announced: Winter 2016Disease Type: Duchenne Muscular Dystrophy (DMD)“Michael Rudnicki is working on a series of experiments to characterize the nature of muscle stem cell defects in a resea...”
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DMD - Alessandra Sacco, Ph.D.
Grant Announced: Winter 2016Disease Type: Duchenne Muscular Dystrophy (DMD)““I have been studying skeletal muscle for 15 years and I have a long-term interest in Duchenne Muscular Dystrophy,” ...”
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DMD - Melissa Spencer, Ph.D.
Grant Announced: Winter 2016Disease Type: Duchenne Muscular Dystrophy (DMD)“Melissa Spencer is working on studies designed to support the development of osteopontin inhibitors to treat Duchenne mu...”
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ALS - Kim Staats, Ph.D.
Grant Announced: Winter 2016Disease Type: Amyotrophic Lateral Sclerosis (ALS)““Our work focuses on the mechanisms that take place during motor neuron loss,” Kim Staats says. “In particular it ...”
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nemaline myopathy - Tamar Sztal, Ph.D.
Grant Announced: Winter 2016Disease Type: Endocrine Myopathies““My previous research has focused mainly on characterizing the pathology of the disease and understanding how this cau...”
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ALS - Christine Vande Velde, Ph.D.
Grant Announced: Winter 2016Disease Type: Amyotrophic Lateral Sclerosis (ALS)““Understanding the basic cell biological mechanisms that have become defective and lead to ALS, and thus serve as ther...”