
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.
Meet the Researchers
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Aaron Beedle, Ph.D.
Funded: 02/01/17 through 01/31/20Grant Announced: Winter 2017Disease Type: Limb-Girdle Muscular Dystrophy (LGMD)“Aaron Beedle and colleagues are working to determine whether drugs that target a common intracellular pathway for cell g...”
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Vijayendran Chandran, Ph.D.
Grant Announced: Winter 2017Disease Type: Friedreich's Ataxia (FA)““Molecular biomarkers are important tools in assessing clinical trial outcomes, measuring disease progression, and eva...”
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Manuela Corti, Ph.D.
Funded: 02/01/17 through 01/31/20Grant Announced: Winter 2017Disease Type: Friedreich's Ataxia (FA)“Manuela Corti aims to optimize gene therapy approaches for replacing frataxin in a mouse model of Friedreich’s ataxia,...”
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Charles Emerson, Ph.D.
Grant Announced: Winter 2017Disease Type: Facioscapulohumeral Muscular Dystrophy (FSH, FSHD)““MDA supports research innovation needed to create breakthroughs for therapeutic development, as well as brings togeth...”
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Darin Falk, Ph.D.
Grant Announced: Winter 2017Disease Type: Metabolic Myopathies“Although a therapy exists for Pompe disease, Darin Falk is working to further enhance treatment efficacy, patient outcom...”
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Mohamed Farah, Ph.D.
Grant Announced: Winter 2017Disease Type: Amyotrophic Lateral Sclerosis (ALS)““Without this grant and previous funding from MDA, we would not have been able to work on motor neuron disease,” Moh...”
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Laxman Gangwani, Ph.D.
Grant Announced: Winter 2017Disease Type: Spinal Muscular Atrophy (SMA)“Laxman Gangwani’s MDA-supported work could lead to treatments for spinal muscular atrophy and other related neuromuscu...”
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Fen-Biao Gao, Ph.D.
Grant Announced: Winter 2017Disease Type: Amyotrophic Lateral Sclerosis (ALS)““Research in the C9ORF72 ALS field is moving forward rapidly,” Fen-Biao Gao says. “We are hopeful that collective ...”
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Emanuela Gussoni, Ph.D.
Grant Announced: Winter 2017Disease Type: Duchenne Muscular Dystrophy (DMD)““I have met children affected by Duchenne muscular dystrophy,” Emanuela Gussoni says. “Helping them to overcome th...”
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Scott Harper, Ph.D.
Funded: 02/01/17 through 01/31/20Grant Announced: Winter 2017Disease Type: Facioscapulohumeral Muscular Dystrophy (FSH, FSHD)““Ten years ago, there was no consensus on the underlying causes of FSHD, we didn't have validated models that express...”
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LGMD and Miyoshi myopathy – Marshall Hogarth, Ph.D.
Grant Announced: Winter 2017Disease Type: Limb-Girdle Muscular Dystrophy (LGMD)““There is still considerable unmet need in understanding dysferlinopathy, and there are no treatment options available...”
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Jie Jiang, Ph.D.
Grant Announced: Winter 2017Disease Type: Amyotrophic Lateral Sclerosis (ALS)“"ALS is one of the most devastating diseases I have ever seen,” Jie Jiang says. “Sadly, no medication is available t...”
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Henry Kaminski, M.D.
Grant Announced: Winter 2017Disease Type: Myasthenia Gravis (MG)““Patients with myasthenia gravis should be encouraged with the improvements in the quality and quantity of basic, tran...”
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Tejvir Khurana, M.D., Ph.D.
Grant Announced: Winter 2017Disease Type: Duchenne Muscular Dystrophy (DMD)““A major inspiration for me to study Duchenne muscular dystrophy and try and develop therapeutic strategies for the di...”
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Kleopas Kleopa, M.D.
Grant Announced: Winter 2017Disease Type: Charcot-Marie-Tooth Disease (CMT)““All of the inherited neuropathies remain incurable despite the progress made in the last couple of decades in underst...”
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Albert La Spada, M.D., Ph.D.
Grant Announced: Winter 2017Disease Type: Spinal-Bulbar Muscular Atrophy (SBMA)““While some of these disorders such as spinal-bulbar muscular atrophy are rare, inherited disorders enable researchers...”
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Nigel Laing, Ph.D.
Grant Announced: Winter 2017Disease Type: Metabolic Myopathies“Nigel Laing and colleagues are conducting preclinical evaluation of two similar treatment approaches for McArdle disease...”
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Eduardo Balsa Martinez, Ph.D.
Grant Announced: Winter 2017Disease Type: Mitochondrial Myopathies (MM)““I hope my work will have a positive impact on the mission of MDA by providing the basis for the development of future...”
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James Novak, Ph.D.
Grant Announced: Winter 2017Disease Type: Duchenne Muscular Dystrophy (DMD)““My desire and motivation to pursue a research career investigating therapeutic interventions for Duchenne muscular dy...”
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Maria Pennuto, Ph.D.
Grant Announced: Winter 2017Disease Type: Spinal-Bulbar Muscular Atrophy (SBMA)““Support from MDA allows us to further our knowledge on disease mechanisms, an aspect that is crucial for our understa...”
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Alexander Polster, Ph.D.
Grant Announced: Winter 2017Disease Type: Endocrine Myopathies““Improvements in treatment of the muscle channelopathies are on the horizon,” Alexander Polster says. “Recent stud...”
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Araya Puwanant, M.D.
Funded: 02/01/17 through 01/31/20Grant Announced: Winter 2017Disease Type: Myotonic Dystrophy (DM)“Araya Puwanant aims to evaluate the use of a simple muscle imaging technique in individuals with type 1 or type 2 myoton...”
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Mattia Quattrocelli, Ph.D.
Grant Announced: Winter 2017Disease Type: Duchenne Muscular Dystrophy (DMD)““The path from refined biological models to novel therapeutic strategies is always extremely difficult, particularly i...”
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Eric Shoubridge, Ph.D.
Funded: 02/01/17 through 01/31/20Grant Announced: Winter 2017Disease Type: Amyotrophic Lateral Sclerosis (ALS)““ALS is a devastating disease for which there is no known cure,” Eric Shoubridge says. “Investigating the rarer ca...”
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Erik Storkebaum, Ph.D.
Grant Announced: Winter 2017Disease Type: Charcot-Marie-Tooth Disease (CMT)““The molecular mechanisms underlying Charcot-Marie-Tooth disease and ALS are poorly understood, and both diseases are ...”
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Maurice Swanson, Ph.D.
Grant Announced: Winter 2017Disease Type: Myotonic Dystrophy (DM)“Maurice Swanson is working to generate and characterize new research models for both type 1 and type 2 myotonic dystroph...”
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Lori Wallrath, Ph.D.
Funded: 02/01/17 through 01/31/20Grant Announced: Winter 2017Disease Type: Congenital Muscular Dystrophy (CMD)““Research on rare types of muscular dystrophy is needed because they are currently not well understood and therapies d...”
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Jiou Wang, M.D., Ph.D.
Grant Announced: Winter 2017Disease Type: Amyotrophic Lateral Sclerosis (ALS)“"Despite increasing public attention to finding a cure, the devastating motor neuron degenerative disease ALS remains a ...”
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Thurman Wheeler, M.D.
Funded: 02/01/17 through 01/31/20Grant Announced: Winter 2017Disease Type: Myotonic Dystrophy (DM)