
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 - Spring 2014
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CMT - Anthony Antonellis, Ph.D.
Grant Announced: Spring 2014Disease Type: Charcot-Marie-Tooth Disease (CMT)“Anthony Antonellis is studying how mutations in genes for certain enzymes lead abnormalities in CMT-affected cells. Char...”
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ALS - Stanley Appel, M.D.
Grant Announced: Spring 2014Disease Type: Amyotrophic Lateral Sclerosis (ALS)“Stanley Appel is studying how the immune system behaves in ALS. Amyotrophic Lateral Sclerosis (ALS)”
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CMT - Elisabetta Babetto, Ph.D.
Grant Announced: Spring 2014Disease Type: Charcot-Marie-Tooth Disease (CMT)“Elisabetta Babetto will study whether manipulating the PHR1 protein pathway helps to preserve nerve fibers in mice with ...”
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Muscle disease - Mariah Baker, Ph.D.
Grant Announced: Spring 2014Disease Type: Becker Muscular Dystrophy (BMD)“Mariah Baker will investigate how calcium is released in muscle cells, which may lead to treatment for a variety of diso...”
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MMD/DM - Andrew Berglund, Ph.D.
Grant Announced: Spring 2014Disease Type: Myotonic Dystrophy (DM)“Andrew Berglund is investigating whether changing the shape of abnormal genetic material in type 1 or type 2 MMD can red...”
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MM - David Chan, M.D., Ph.D.
Grant Announced: Spring 2014Disease Type: Mitochondrial Myopathies (MM)“David Chan will study defects in the behavior of mitochondria, which produce energy in cells, in mice with mitochondrial...”
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CMT - Gavriel David, M.D., Ph.D.
Grant Announced: Spring 2014Disease Type: Charcot-Marie-Tooth Disease (CMT)“Gavriel David is investigating calcium overload in CMT-affected nerve cells. Shown here is a mouse nerve fiber, with an ...”
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DMD - Dongsheng Duan, Ph.D.
Grant Announced: Spring 2014Disease Type: Duchenne Muscular Dystrophy (DMD)“Dongsheng Duan will maintain a tissue bank for investigators studying DMD. Duchenne Muscular Dystrophy (DMD)”
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DMD - Nicoletta Filigheddu, Ph.D.
Grant Announced: Spring 2014Disease Type: Duchenne Muscular Dystrophy (DMD)“Nicoletta Filigheddu will look at the effects of a hormone called ghrelin in dystrophin-deficient mice, which have a dis...”
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ALS - Aaron Gitler, Ph.D.
Grant Announced: Spring 2014Disease Type: Amyotrophic Lateral Sclerosis (ALS)“Aaron Gitler is investigating how profilin 1 gene mutations cause ALS, with the hope that understanding this phenomenon ...”
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DMD - Darek Gorecki, Ph.D.
Grant Announced: Spring 2014Disease Type: Duchenne Muscular Dystrophy (DMD)“Image A shows the receptors that Gorecki and colleagues will try to block in green, with muscle fibers indicated by Ms. ...”
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Nemaline Myopathy - Henk Granzier, Ph.D.
Grant Announced: Spring 2014Disease Type: Endocrine Myopathies“Henk Granzier is shown here with a microscope-based system he uses to measure the mechanical properties of nebulin molec...”
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ALS - Maurizio Grimaldi, M.D., Ph.D.
Grant Announced: Spring 2014Disease Type: Amyotrophic Lateral Sclerosis (ALS)“Maurizio Grimaldi is developing new molecules that may protect nerve cells in ALS. Amyotrophic Lateral Sclerosis (ALS)”
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ALS - Amanda Haidet-Phillips, Ph.D.
Grant Announced: Spring 2014Disease Type: Amyotrophic Lateral Sclerosis (ALS)“Amanda Haidet-Phillips is investigating how upper motor neurons are lost in ALS. Here, upper motor neurons are shown in ...”
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Muscle disease - Stephen Hauschka, Ph.D.
Grant Announced: Spring 2014Disease Type: Becker Muscular Dystrophy (BMD)“Stephen Hauschka is developing on-off switches to use in gene transfer therapies for muscle disorders. Muscular Dystroph...”
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ALS - Muralidhar Hegde, Ph.D.
Grant Announced: Spring 2014Disease Type: Amyotrophic Lateral Sclerosis (ALS)“Muralidhar Hegde is studying whether inadequate DNA repair by a protein called TDP43 is involved in causing ALS. Amyotro...”
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MMD/DM - Bernard Jasmin, Ph.D.
Grant Announced: Spring 2014Disease Type: Myotonic Dystrophy (DM)“Bernard Jasmin will examine the functions of a protein called staufen 1 with an eye to developing a treatment for type 1...”
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CMT - Nivedita Jerath, M.D.
Grant Announced: Spring 2014Disease Type: Charcot-Marie-Tooth Disease (CMT)“Nivedita Jerath is studying driving ability in people with type 1A CMT. Charcot-Marie-Tooth Disease (CMT)”
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SMA - Stephen Kolb, M.D., Ph.D.
Grant Announced: Spring 2014Disease Type: Spinal Muscular Atrophy (SMA)“Stephen Kolb is determining how best to measure SMA progression during the first two years of life so that experimental ...”
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SMA - Rashmi Kothary, Ph.D.
Grant Announced: Spring 2014Disease Type: Spinal Muscular Atrophy (SMA)“Rashmi Kothary (center, standing) is investigating a new therapeutic avenue for SMA. Spinal Muscular Atrophy (SMA)”
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Muscle disease - Shihuan Kuang, Ph.D.
Grant Announced: Spring 2014Disease Type: Becker Muscular Dystrophy (BMD)“Shihuan Kuang is investigating a technique that may improve stem cell transplantation as a treatment for muscular dystro...”
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CMS/LGMD/PP - Jocelyn Laporte, Ph.D.
Grant Announced: Spring 2014Disease Type: Congenital Myasthenic Syndromes (CMS)“Jocelyn Lapote (left) is studying whether drugs that alter calcium flow in cells can be used to treat congenital myasthe...”
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CMD/LGMD - Maria Chiara Manzini, Ph.D.
Grant Announced: Spring 2014Disease Type: Congenital Muscular Dystrophy (CMD)“Maria Chiara Manzini is studying how mutations that cause human CMD and LGMD affect muscle function in zebrafish, conduc...”
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CMD/LGMD - Maria Chiara Manzini, Ph.D.
Grant Announced: Spring 2014Disease Type: Congenital Muscular Dystrophy (CMD)“Maria Chiara Manzini is studying how mutations that cause human CMD and LGMD affect muscle function in zebrafish, conduc...”
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LEMS - Stephen Meriney, Ph.D.
Grant Announced: Spring 2014Disease Type: Lambert-Eaton Myasthenic Syndrome (LEMS)“Stephen Meriney is shown here with one of his experimental setups, which records nerve-to-muscle signals from mice with ...”
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CMT/DSD - Kelly Monk, Ph.D.
Grant Announced: Spring 2014Disease Type: Charcot-Marie-Tooth Disease (CMT)“Kelly Monk is studying the formation and maintenance of the myelin sheath, an insulating coating that surrounds many ner...”
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DMD - Federica Montanaro, Ph.D.
Grant Announced: Spring 2014Disease Type: Duchenne Muscular Dystrophy (DMD)“Federica Montanaro will investigate whether increasing a signaling pathway called "hedgehog" will help preserve muscle f...”
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MM - Carlos Moraes, Ph.D.
Grant Announced: Spring 2014Disease Type: Mitochondrial Myopathies (MM)“Carlos Moraes (back row, second from left) and his co-workers will investigate the potential of DNA-cutting enzymes to t...”
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SMA - Lyndsay Murray, Ph.D.
Grant Announced: Spring 2014Disease Type: Spinal Muscular Atrophy (SMA)“Lyndsay Murray (front) will investigate a potential new strategy for protecting nerve cells that carry SMA-causing abnor...”
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DMD - Terence Partridge, Ph.D.
Grant Announced: Spring 2014Disease Type: Duchenne Muscular Dystrophy (DMD)“Terence Partridge is studying an exon-skipping compound designed to treat DMD that appears to enter the heart in additio...”
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MM - Catarina Quinzii, M.D
Grant Announced: Spring 2014Disease Type: Mitochondrial Myopathies (MM)“Catarina Quinzii will investigate how abnormalities in a protein called RMND1 cause a mitochondrial disorder. Mitochondr...”
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DMD - Erica Reeves, Ph.D.
Grant Announced: Spring 2014Disease Type: Duchenne Muscular Dystrophy (DMD)“Erica Reeves at ReveraGen BioPharma is testing VBP15, an experimental drug for DMD. Duchenne Muscular Dystrophy (DMD)”
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CMT - Michael Shy, M.D.
Grant Announced: Spring 2014Disease Type: Charcot-Marie-Tooth Disease (CMT)“Michael Shy plans to conduct a clinical trial of an experimental treatment in patients with CMT1B. Charcot-Marie-Tooth D...”
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CMT - Michael Shy, M.D.
Grant Announced: Spring 2014Disease Type: Charcot-Marie-Tooth Disease (CMT)“Michael Shy plans to conduct a clinical trial of an experimental treatment in patients with CMT1B. Charcot-Marie-Tooth D...”
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CMT - Ludo Van Den Bosch, Ph.D.
Grant Announced: Spring 2014Disease Type: Charcot-Marie-Tooth Disease (CMT)“Ludo Van Den Bosch is investigating the possible use of a compound known as an HDAC6 inhibitor in treating type 2F CMT a...”
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DMD - Da-Zhi Wang, Ph.D.
Grant Announced: Spring 2014Disease Type: Duchenne Muscular Dystrophy (DMD)“Da-Zhi Wang is seeking to find out whether muscle regeneration can be improved in DMD. Duchenne Muscular Dystrophy (DMD)”
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EDMD - Howard Worman, M.D.
Grant Announced: Spring 2014Disease Type: Emery-Dreifuss Muscular Dystrophy (EDMD)“Howard Worman will study a possible new treatment for EDMD in mice with a disorder mimicking the human disease. Emery-Dr...”
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MMD/DM - Steven Zimmerman, Ph.D.
Grant Announced: Spring 2014Disease Type: Myotonic Dystrophy (DM)“Steven Zimmerman is developing an experimental drug for type 1 MMD. Myotonic Muscular Dystrophy (MMD)”