
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.
Summer Grants 2017
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DM – Andrew Berglund, Ph.D.
Funded: 08/01/17 through 07/31/20Grant Announced: Summer 2017Disease Type: Myotonic Dystrophy (DM)“I think the myotonic dystrophy field has made great gains in understanding the mechanisms of this disease, and there a n...”
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Nemaline Myopathy – Jordan Blondelle, Ph.D.
Funded: 08/01/17 through 07/31/20Grant Announced: Summer 2017Disease Type: Endocrine Myopathies“My focus is to better understand the role that Cullin-3-related protein degradation may play in the pathological mechani...”
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DMD – Dean Burkin, Ph.D.
Funded: 08/01/17 through 07/31/20Grant Announced: Summer 2017Disease Type: Duchenne Muscular Dystrophy (DMD)“I have received other MDA grants which have been critical to these studies,” Dean Burkin says. “This new grant will ...”
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Mitochondrial Myopathy, DMD – Gino Cortopassi, Ph.D.
Funded: 08/01/17 through 07/31/20Grant Announced: Summer 2017Disease Type: Mitochondrial Myopathies (MM)“We believe that this drug, already approved for use in humans, will cure mitochondrial disease in animals,” Gino Corto...”
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DM – Auinash Kalsotra, Ph.D.
Funded: 08/01/17 through 07/31/20Grant Announced: Summer 2017Disease Type: Myotonic Dystrophy (DM)“I feel that the DM field is making impressive strides in deciphering the exact pathogenic mechanisms unleashed by the tr...”
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FSHD – Angela Lek, Ph.D.
Funded: 08/01/17 through 07/31/20Grant Announced: Summer 2017Disease Type: Facioscapulohumeral Muscular Dystrophy (FSH, FSHD)“Without the support of MDA, young scientists like myself will not have the means to focus and shine a spotlight on rare ...”
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SBMA – Andrew Lieberman, M.D., Ph.D.
Funded: 08/01/17 through 07/31/20Grant Announced: Summer 2017Disease Type: Spinal-Bulbar Muscular Atrophy (SBMA)“There are no available disease-modifying treatments for SBMA,” Andrew Lieberman says. “Grants from the MDA provide c...”
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All neuromuscular diseases (NMDs) – James Lupski, M.D., Ph.D., D.Sc. (hon)
Funded: 08/01/17 through 07/31/20Grant Announced: Summer 2017Disease Type: Congenital Muscular Dystrophy (CMD)“Obtaining a molecular diagnosis is extremely important for patients and families with NMDs,” James Lupski says, “as ...”
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DMD – Joseph Metzger, Ph.D.
Funded: 08/01/17 through 07/31/20Grant Announced: Summer 2017Disease Type: Duchenne Muscular Dystrophy (DMD)“Numerous inherited and acquired cardiac diseases are directly linked to deficits in dystrophin protein, including, for e...”
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ALS – Timothy Miller, M.D., Ph.D.
Funded: 08/01/17 through 07/31/20Grant Announced: Summer 2017Disease Type: Amyotrophic Lateral Sclerosis (ALS)“For C9ORF72 ALS, dipeptide repeat protein (DPR) aggregation seems to be uncorrelated with degeneration in autopsy tissue...”
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DMD – Philippe Moullier, M.D., Ph.D.
Funded: 08/01/17 through 07/31/20Grant Announced: Summer 2017Disease Type: Duchenne Muscular Dystrophy (DMD)“Overall,” says Philippe Moullier, “the results of this program will provide important preclinical proof-of-concept t...”
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ALS – Udai Pandey, Ph.D.
Funded: 08/01/17 through 07/31/20Grant Announced: Summer 2017Disease Type: Amyotrophic Lateral Sclerosis (ALS)“Udai Pandey says his team’s small molecule screening project could help identify drugs that might be useful for testin...”
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DMD – Feng Yue, Ph.D.
Funded: 08/01/17 through 07/31/20Grant Announced: Summer 2017Disease Type: Duchenne Muscular Dystrophy (DMD)“Our study will first provide critical insights into the mechanistic basis by which direct inactivation of PTEN in muscle...”