
Innovations in Science
We're accelerating the delivery of treatments and cures.
Summer Grants 2019
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MM - Antonio Barrientos, PhD
Funded: 08/01/19 through 07/31/22Grant Announced: Summer 2019Disease Type: Mitochondrial Myopathies (MM)“Better understanding of the cellular and mitochondrial processes leading to COX assembly is expected to have a significa...”
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SMA - Arthur Burghes, PhD
Funded: 08/01/19 through 07/31/21Grant Announced: Summer 2019Disease Type: Spinal Muscular Atrophy (SMA)“In this proposal we aim to identify the genes that modify the severity of SMA and the changes in them that cause modific...”
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CMD - Dean Burkin, PhD
Funded: 08/01/19 through 07/31/22Grant Announced: Summer 2019Disease Type: Congenital Muscular Dystrophy (CMD)“We hope this laminin-enhancing small molecule will slow muscle pathology and improve muscle strength in a mouse model of...”
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FSHD - Yi-Wen Chen, DVM, PhD
Funded: 08/01/19 through 07/31/21Grant Announced: Summer 2019Disease Type: Facioscapulohumeral Muscular Dystrophy (FSH, FSHD)“We hope that we can demonstrate good bioavailability of the antisense oligonucleotide (ASO) therapy in muscle cells and ...”
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LGMD - Martin Childers, PhD, DO
Funded: 08/01/19 through 07/31/20Grant Announced: Summer 2019Disease Type: Limb-Girdle Muscular Dystrophy (LGMD)“Martin Childers, PhD, DO, chief medical officer at Asklepios BioPharmaceutical Inc. in North Carolina, was awarded an MD...”
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DMD - Peter Currie, PhD
Funded: 08/01/19 through 07/31/22Grant Announced: Summer 2019Disease Type: Duchenne Muscular Dystrophy (DMD)“Peter Currie, PhD, professor of Medicine, Nursing, Health Sciences, and Developmental and Regenerative Biology at Monash...”
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ALS - Sandrine Da Cruz, PhD
Funded: 08/01/19 through 07/31/22Grant Announced: Summer 2019Disease Type: Amyotrophic Lateral Sclerosis (ALS)“This work will provide critical insight for elucidating the mechanisms regulating protein aggregation and cell-to-cell s...”
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CMT - Gerald W. Dorn, II, MD
Funded: 08/01/19 through 07/31/21Grant Announced: Summer 2019Disease Type: Charcot-Marie-Tooth Disease (CMT)“There is currently no disease-modifying treatment for CMT2A; mitofusin agonists may be the first. This is an orphan dise...”
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ALS - Jone Lopez Erauskin, PhD
Funded: 08/01/19 through 07/31/22Grant Announced: Summer 2019Disease Type: Amyotrophic Lateral Sclerosis (ALS)“I expect to determine how stathmin-2 provides a crucial role in the maintenance and regeneration of the neuromuscular ju...”
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DMD - James Ervasti, PhD
Funded: 08/01/19 through 07/31/22Grant Announced: Summer 2019Disease Type: Duchenne Muscular Dystrophy (DMD)“While there has been tremendous progress in the advancement of therapies to treat DMD patients, we still have not cured ...”
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DMD - Michael Hicks, PhD
Funded: 08/01/19 through 07/31/22Grant Announced: Summer 2019Disease Type: Duchenne Muscular Dystrophy (DMD)“Understanding how muscle stem cells interact with their niche is hugely important. It will help us understand how muscle...”
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DM - Melissa Hinman, PhD
Funded: 08/01/19 through 07/31/22Grant Announced: Summer 2019Disease Type: Myotonic Dystrophy (DM)“Although digestive symptoms affect quality of life, few studies have focused on understanding their causes or developing...”
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DMD - Changwon Kho, PhD
Funded: 08/01/19 through 07/31/22Grant Announced: Summer 2019Disease Type: Duchenne Muscular Dystrophy (DMD)“Given the high burden of cardiomyopathy in DMD patients, it is vital to identify the pathophysiological mechanisms in pl...”
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CMD, LGMD - Monkol Lek, PhD
Funded: 08/01/19 through 07/31/22Grant Announced: Summer 2019Disease Type: Limb-Girdle Muscular Dystrophy (LGMD)“My focus in the genetics of neuromuscular diseases is to discover novel disease genes [and] mechanisms and build resourc...”
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IBM - Thomas Lloyd, MD, PhD
Funded: 08/01/19 through 07/31/22Grant Announced: Summer 2019Disease Type: Inclusion-Body Myositis (IBM)“We expect that we will be able to use this model to better understand the role that inflammation plays in developing IBM...”
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FA - Jordi Magrane, PhD
Funded: 08/01/19 through 07/31/22Grant Announced: Summer 2019Disease Type: Friedreich's Ataxia (FA)“Jordi Magrane, PhD, associate professor of Neuroscience at Weill Cornell Medical College in New York, was awarded an MDA...”
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IBM - Bradley Olwin, PhD
Funded: 08/01/19 through 07/31/22Grant Announced: Summer 2019Disease Type: Inclusion-Body Myositis (IBM)“Bradley Olwin, PhD, professor of Molecular, Cellular, and Developmental Biology at the University of Colorado Boulder, w...”
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CMT, MM, other NMDs - Wolfgang Pernice, PhD
Funded: 08/01/19 through 07/31/22Grant Announced: Summer 2019Disease Type: Charcot-Marie-Tooth Disease (CMT)“Our research aims to develop unbiased experimental strategies and tools to systematically address critical roadblocks in...”
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ALS - Jeffrey D. Rothstein, MD, PhD
Funded: 08/01/19 through 07/31/22Grant Announced: Summer 2019Disease Type: Amyotrophic Lateral Sclerosis (ALS)“Jeffrey D. Rothstein, MD, PhD, director of the Brain Science Institute and director of the Robert Packard Center for ALS...”
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SMA - Ashlyn Spring, PhD
Funded: 08/01/19 through 07/31/22Grant Announced: Summer 2019Disease Type: Spinal Muscular Atrophy (SMA)“Ashlyn Spring, PhD, a postdoctoral fellow in Biological and Genome Sciences at the University of North Carolina at Chape...”
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CMT, SMA - Charlotte Sumner, MD
Funded: 08/01/19 through 07/31/22Grant Announced: Summer 2019Disease Type: Charcot-Marie-Tooth Disease (CMT)“Charlotte Sumner, MD, professor of Neurology and Neuroscience at Johns Hopkins University School of Medicine in Baltimor...”
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SBMA - Alireza Mashaghi Tabari, PhD
Funded: 08/01/19 through 07/31/22Grant Announced: Summer 2019Disease Type: Spinal-Bulbar Muscular Atrophy (SBMA)“To date, not many labs have probed the underlying nature of genetically inherited diseases using optical tweezer techniq...”
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ALS - Davide Trotti, PhD
Funded: 08/01/19 through 07/31/22Grant Announced: Summer 2019Disease Type: Amyotrophic Lateral Sclerosis (ALS)“Davide Trotti, PhD, professor of Neuroscience at Thomas Jefferson University in Philadelphia, was awarded an MDA researc...”
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DMD - Courtney Young, PhD
Funded: 08/01/19 through 07/31/22Grant Announced: Summer 2019Disease Type: Duchenne Muscular Dystrophy (DMD)“This work will further advance pre-clinical development of our gene editing therapy for Duchenne. Assessing the immune r...”
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DMD, SMA - Damiano Zanotto, PhD
Funded: 08/01/19 through 07/31/21Grant Announced: Summer 2019Disease Type: Duchenne Muscular Dystrophy (DMD)“The study of patients’ walking patterns in their day-to-day life can help researchers and clinicians better understand...”