
Innovations in Science
We're accelerating the delivery of treatments and cures.
Summer Grants 2020
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Clinical Procedures to Support Research (CAPTURE) - Michael Benatar, M.D., Ph.D.
Funded: 07/01/20 through 06/30/23Grant Announced: Summer 2020Disease Type: Amyotrophic Lateral Sclerosis (ALS)“A holy-grail of physician-investigators engaged in both ALS patient care and research has been the efficient and effecti...”
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Generation of large dystrophins in muscle via AAV vector delivery - Jeffrey Chamberlain, PhD
Funded: 05/01/20 through 04/30/22Grant Announced: Summer 2020Disease Type: Duchenne Muscular Dystrophy (DMD)“Our new approach will continue development of a novel approach to delivering larger, possibly up to full-length, dystrop...”
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Clinical Research in ALS (CRiALS): A clinical database and specimen biobank - Jonathan Glass, MD
Funded: 01/01/20 through 12/31/20Grant Announced: Summer 2020Disease Type: Amyotrophic Lateral Sclerosis (ALS)“There is an enormous need for a better understanding of the clinical, demographic, and genetic features that underlie th...”
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Machine Learning of Neuromuscular Disease Gait Parameters from Single Sensors - Erik Henricson, PhD
Funded: 01/01/20 through 12/31/22Grant Announced: Summer 2020Disease Type: Charcot-Marie-Tooth Disease (CMT)“The overall goal of this project is to develop new and innovative mobile device-compatible methodologies based on low-co...”
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Cardiac Regenerative Model for MD - Qi Lu, MD, PhD
Funded: 01/01/20 through 12/31/21Grant Announced: Summer 2020Disease Type: Duchenne Muscular Dystrophy (DMD)“Cardiomyopathy is a prominent feature of many neuromuscular diseases including Duchenne muscular dystrophy (DMD).”
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Statistical analyses of the ALS COSMOS, ARREST ALS, and ARREST Control studies - Hiroshi Mitsumoto, MD, DSci
Funded: 01/01/20 through 12/31/20Grant Announced: Summer 2020Disease Type: Amyotrophic Lateral Sclerosis (ALS)“We will conduct crucial statistical analyses to uncover ALS risk factors and the cause of ALS.”
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ALS Platform Trial: Accelerating the path to effective treatments - Sabrina Paganoni, MD, PhD
Funded: 04/01/20 through 03/31/23Grant Announced: Summer 2020Disease Type: Amyotrophic Lateral Sclerosis (ALS)“To accelerate therapy development, we designed the first Platform Trial for ALS in the world. Traditionally, each trial ...”
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Validating an AAV gene therapeutic strategy for ALS-FTD - Jeffrey Rothstein, MD, PhD
Funded: 03/01/20 through 02/28/21Grant Announced: Summer 2020Disease Type: Amyotrophic Lateral Sclerosis (ALS)“Recent studies in human disease support the notion that loss of such TDP43 function is compromised as an early event in ...”
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Sequence Analysis of Myotonic Dystrophy - Charles Thornton, MD
Funded: 03/01/20 through 02/28/21Grant Announced: Summer 2020Disease Type: Myotonic Dystrophy (DM)“With drug development for myotonic dystrophy 1 (DM1) progressing rapidly, "access to genetic testing could become a sign...”
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Data Resource for CMT Genomics - Stephan Zuchner, MD, PhD
Funded: 01/01/21 through 12/31/23Grant Announced: Summer 2020Disease Type: Charcot-Marie-Tooth Disease (CMT)“An open Charcot-Marie-Tooth genetics data resource could help expedite CMT research, from gene identification to drug di...”