
Innovations in Science
We're accelerating the delivery of treatments and cures.
Summer Grants 2018
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CMT – Bogdan Karl Beirowski, MD, PhD
Funded: 08/01/18 through 07/31/21Grant Announced: Summer 2018Disease Type: Charcot-Marie-Tooth Disease (CMT)“Axon degeneration is a central component of irreversible neurological disability in many neuromuscular diseases. There i...”
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CMT – Alessandra Bolino, PhD
Funded: 08/01/18 through 07/31/21Grant Announced: Summer 2018Disease Type: Charcot-Marie-Tooth Disease (CMT)“Our research is aimed at unravelling how myelination is regulated in the PNS and how perturbation of these mechanisms ca...”
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Pompe and LGMD – Dr. Samya Chakravorty, PhD
Funded: 08/01/18 through 07/31/21Grant Announced: Summer 2018Disease Type: Limb-Girdle Muscular Dystrophy (LGMD)“This work will specify what proportion of LGMD multi-genic cases can reach a molecular diagnosis and get enrolled in cli...”
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LGMD – Dr. Peter Davies, PhD
Funded: 08/01/18 through 07/31/21Grant Announced: Summer 2018Disease Type: Limb-Girdle Muscular Dystrophy (LGMD)“Given our past successes with calpains -1 and -2, we are now applying the same structure-guided approaches to study the ...”
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Congenital Myopathy– Dr. Frances Evesson, PhD
Funded: 08/01/18 through 07/31/21Grant Announced: Summer 2018Disease Type: Congenital Myopathies“I am studying the pathology of a newly identified form of congenital myopathy caused by variants in an enzyme called PYR...”
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FSHD – Lawrence James Hayward, MD, PhD
Funded: 08/01/18 through 07/31/21Grant Announced: Summer 2018Disease Type: Facioscapulohumeral Muscular Dystrophy (FSH, FSHD)“This condition causes lifelong disability yet is under-recognized by clinicians. What has been amazing to me is the coll...”
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Mitochondrial Myopathy – Michio Hirano, MD
Funded: 08/01/18 through 07/31/21Grant Announced: Summer 2018Disease Type: Mitochondrial Myopathies (MM)
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DMD – Shama Rajan Iyer, PhD
Funded: 08/01/18 through 07/31/21Grant Announced: Summer 2018Disease Type: Duchenne Muscular Dystrophy (DMD)“I began my research in skeletal muscle biology by developing biologic coatings and scaffolds to improve skeletal muscle ...”
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Myopathy and ALS – Dr. Virginia Kimonis, MD, MRCP
Funded: 08/01/18 through 07/31/20Grant Announced: Summer 2018Disease Type: Amyotrophic Lateral Sclerosis (ALS)“We hope that inhibition of VCP will improve the typical disease pathology in the muscle and spinal cord in the cell and ...”
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SMA – Rashmi Kothary, PhD
Funded: 08/01/18 through 07/31/21Grant Announced: Summer 2018Disease Type: Spinal Muscular Atrophy (SMA)“There has to be great optimism about the emerging therapies for the treatment of SMA. There has been tremendous progress...”
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Centronuclear myopathies – Jocelyn Laporte, PhD
Funded: 08/01/18 through 07/31/21Grant Announced: Summer 2018Disease Type: Centronuclear myopathies“I hope people affected by neuromuscular diseases feel that researchers are indeed working hard and in the best ethical m...”
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ALS – Christopher McDermott, MBChB, FRCP, PhD
Funded: 03/01/18 through 12/31/18Grant Announced: Summer 2018Disease Type: Amyotrophic Lateral Sclerosis (ALS)“There have been conflicting studies with regards to whether diaphragm pacing is a good thing to offer people living with...”
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ALS – Danilo Medinas, PhD
Funded: 08/01/18 through 07/31/21Grant Announced: Summer 2018Disease Type: Amyotrophic Lateral Sclerosis (ALS)
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SMA – Jacqueline Montes, PT, EdD, NCS
Funded: 04/01/18 through 04/30/21Grant Announced: Summer 2018Disease Type: Spinal Muscular Atrophy (SMA)
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Mitochondrial Myopathies – Carlos T. Moraes, PhD
Funded: 08/01/18 through 08/31/21Grant Announced: Summer 2018Disease Type: Mitochondrial Myopathies (MM)“There are no treatments for mitochondrial diseases. The unique genetics of mitochondria offer some opportunities for gen...”
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DMD – Pura Muñoz-Canoves, PhD
Funded: 08/01/18 through 07/31/21Grant Announced: Summer 2018Disease Type: Duchenne Muscular Dystrophy (DMD)“New emerging advances in basic and applied science may open definitive doors to cures.”
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CMD & Congenital Myopathies – Francesco Muntoni, MD
Funded: 08/01/18 through 07/30/21Grant Announced: Summer 2018Disease Type: Congenital Myopathies“Research focused on identification of the genetic basis of other neuromuscular disorders has paved the road toward devel...”
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ALS – Katharine Nicholson, MD
Funded: 08/01/18 through 07/30/20Grant Announced: Summer 2018Disease Type: Amyotrophic Lateral Sclerosis (ALS)“By studying a population at risk for developing ALS, this project has the potential to further our understanding of unde...”
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MG – Kevin C. O’Connor, PhD
Funded: 08/01/18 through 07/31/21Grant Announced: Summer 2018Disease Type: Myasthenia Gravis (MG)“Overall, this work will further our understanding of how MG initiates and progresses, and uncover fundamental disease me...”
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ALS – Laura P.W. Ranum, PhD
Funded: 08/01/18 through 07/31/21Grant Announced: Summer 2018Disease Type: Amyotrophic Lateral Sclerosis (ALS)““We are at an exciting time in the field of repeat expansion disorders because as a research community we are starting...”
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ALS – Joel Richter, PhD
Funded: 08/01/18 through 07/31/21Grant Announced: Summer 2018Disease Type: Amyotrophic Lateral Sclerosis (ALS)““There are two outcomes we hope for. One outcome is basic understanding of how toxic peptides are generated from C9orf...”
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SBMA – Carlo Rinaldi, MD, PhD
Funded: 08/01/18 through 07/31/21Grant Announced: Summer 2018Disease Type: Spinal-Bulbar Muscular Atrophy (SBMA)“Better understanding of the underlying disease mechanisms, coupled with improvement of gene vector design, therapeutic g...”
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DMD – Dr. Russell Rogers, PhD
Funded: 08/01/18 through 07/31/21Grant Announced: Summer 2018Disease Type: Duchenne Muscular Dystrophy (DMD)“Many cutting-edge approaches to treating DMD are currently under development. Each offers distinct advantages over curre...”
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ALS – Jeffrey D. Rothstein, MD, PhD
Funded: 02/01/18 through 01/31/21Grant Announced: Summer 2018Disease Type: Amyotrophic Lateral Sclerosis (ALS)“Answer ALS is a nationwide consortium that is assembling the largest and most comprehensive ALS dataset to date, in whic...”
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LGMD – Dorianna Sandona, PhD
Funded: 08/01/18 through 07/31/21Grant Announced: Summer 2018Disease Type: Limb-Girdle Muscular Dystrophy (LGMD)“In the last years, researchers understood that to fight orphan diseases the sole possibility is to join forces and share...”
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DMD – Melissa Spencer, PhD
Funded: 08/01/18 through 07/31/21Grant Announced: Summer 2018Disease Type: Duchenne Muscular Dystrophy (DMD)“One of the biggest barriers to success for gene therapies is the ability to deliver the cargo to the tissue of interest....”
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FSHD – Francis Michael Sverdrup, PhD
Funded: 08/01/18 through 07/31/21Grant Announced: Summer 2018Disease Type: Facioscapulohumeral Muscular Dystrophy (FSH, FSHD)“I was inspired to start drug discovery research in FSHD when my daughter was diagnosed with the disease at the age of 10...”
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CMT – Vincent Timmerman, PhD
Funded: 08/01/18 through 07/31/21Grant Announced: Summer 2018Disease Type: Charcot-Marie-Tooth Disease (CMT)“If we can successfully validate our research hypothesis, reducing the expression of HSPB8 in vivo and decreasing the det...”
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ALS – Ludo Van Den Bosch, PhD
Funded: 08/01/18 through 07/31/21Grant Announced: Summer 2018Disease Type: Amyotrophic Lateral Sclerosis (ALS)“Amyotrophic lateral sclerosis (ALS) is a terrible disease, and despite all the research done in the past, patients still...”
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Myotonic Dystrophy – Eric Wang, PhD
Funded: 08/01/18 through 07/31/21Grant Announced: Summer 2018Disease Type: Myotonic Dystrophy (DM)“We are living in an extremely exciting time because of the explosion of technological and conceptual advances, both in t...”