Innovations in Science
We're accelerating the delivery of treatments and cures.
Grantee:
Jocelyn Laporte, Ph.D.
Funded: 9/1/2022 - 8/31/2023
Grant Type:
Idea Award
Disease(s):
Distal myopathies
Grantee:
Genetic Analysis of Myotonic Dystrophy - Charles Thornton, MD
Funded: 3/1/2021 - 2/28/2022
Grant Type:
Restricted Research Grant
Disease(s):
Myotonic dystrophy (DM)
Grantee:
Unbiased lipid screening to reverse ALS Phenotypes in vitro and in vivo - Jeffrey Rothstein, MD, PhD
Funded: 3/1/2021 - 2/28/2022
Grant Type:
Restricted Research Grant
Disease(s):
ALS (amyotrophic lateral sclerosis)
Grantee:
Upper Motor Neuron Onset ALS: Natural History Study - Hiroshi Mitsumoto, MD, DSci
Funded: 1/1/2021 - 12/31/2021
Grant Type:
Restricted Research Grant
Disease(s):
ALS (amyotrophic lateral sclerosis)
Grantee:
Testing a combinatorial therapy of ribitol and gene therapy to treat FKRP-related dystroglycanopathies - Qi Lu, MD, PhD
Funded: 1/1/2021 - 12/31/2021
Grant Type:
Restricted Research Grant
Disease(s):
Limb-girdle muscular dystrophies (LGMD)
Grantee:
Clinical Research in ALS (CRiALS): A clinical database and specimen biobank - Jonathan Glass, MD
Funded: 1/1/2021 - 12/31/2021
Grant Type:
Restricted Research Grant
Disease(s):
ALS (amyotrophic lateral sclerosis)
Grantee:
Identifying novel SMA-causing variants by NGS sequencing of the SMN1 locus – Matthew Avenarius, PhD
Funded: 9/1/2021 - 8/31/2022
Grant Type:
Idea Award
Disease(s):
Spinal muscular atrophy (SMA)
Grantee:
Novel high throughput method to assess micro-dystrophin turnover in muscle – Joseph Metzger, PhD
Funded: 9/1/2021 - 8/31/2022
Grant Type:
Idea Award
Disease(s):
Duchenne muscular dystrophy (DMD)
Grantee:
Assessment of Nocturnal Hypoventilation in Amyotrophic Lateral Sclerosis – Andrew Geronimo, PhD
Funded: 9/1/2021 - 8/31/2022
Grant Type:
Idea Award
Disease(s):
ALS (amyotrophic lateral sclerosis)
Grantee:
Innovative technologies to unravel the complexity of myotonic dystrophy repeats – Massimo Delledonne, MSc, PhD
Funded: 10/1/2021 - 9/30/2022
Grant Type:
Idea Award
Disease(s):
Myotonic dystrophy (DM)
Grantee:
Establishing a network of highly trained and reliable CMT clinical evaluators – Joshua Burns, PhD
Funded: 9/1/2021 - 8/31/2022
Grant Type:
Idea Award
Disease(s):
Charcot-Marie-Tooth disease (CMT)
Grantee:
Jag1 gene therapy in muscular dystrophies – Stefano Biressi, PhD
Funded: 9/1/2021 - 8/31/2022
Grant Type:
Idea Award
Disease(s):
Duchenne muscular dystrophy (DMD)
Grantee:
Measuring cardiopulmonary responses to the 6MWT and peak effort exercise in DMD – Tanja Taivassalo, PhD
Funded: 9/1/2021 - 8/31/2022
Grant Type:
Idea Award
Disease(s):
Duchenne muscular dystrophy (DMD)
Grantee:
Computational Design of Modified Dystrophins – Nick Menhart, PhD
Funded: 9/1/2021 - 8/31/2022
Grant Type:
Idea Award
Disease(s):
Duchenne muscular dystrophy (DMD)
Grantee:
Glucocorticoid receptor and muscle stem cell quiescence – Alexandre Blais, PhD
Funded: 9/1/2021 - 8/31/2022
Grant Type:
Idea Award
Disease(s):
Duchenne muscular dystrophy (DMD)
Grantee:
Re-purposing dimethyl fumarate for the treatment for Duchenne Muscular Dystrophy – Emma Rybalka, PhD
Funded: 9/1/2021 - 8/31/2022
Grant Type:
Idea Award
Disease(s):
Duchenne muscular dystrophy (DMD)
Grantee:
Suppression of COL6 premature termination codons by anticodon-edited tRNAs – Valérie Allamand, PhD
Funded: 10/1/2021 - 9/30/2022
Grant Type:
Idea Award
Disease(s):
Congenital muscular dystrophies (CMD)
Grantee:
The RNA exosome complex in ALS disease pathogenesis – Frederick Arnold, PhD
Funded: 9/1/2021 - 8/31/2024
Grant Type:
Development Grant
Disease(s):
ALS (amyotrophic lateral sclerosis)
Grantee:
Uncovering the Genomic Architecture of Undiagnosed Neuromuscular Disease – Daniel Calame, MD, PhD
Funded: 9/1/2021 - 8/31/2024
Grant Type:
Development Grant
Disease(s):
Congenital muscular dystrophies (CMD)
Grantee:
Targeting Leucyl-tRNA synthetase and autophagy in Duchenne muscular dystrophy – Jae-Sung You, PhD
Funded: 9/1/2021 - 8/31/2023
Grant Type:
Development Grant
Disease(s):
Duchenne muscular dystrophy (DMD)
Grantee:
Identification of modifier gene(s) of spinal muscular atrophy in model mice – Narendra Jha, PhD
Funded: 9/1/2021 - 8/31/2024
Grant Type:
Development Grant
Disease(s):
Spinal muscular atrophy (SMA)
Grantee:
Beneficial impact of Resolvin-D2 on Duchenne Muscular Dystrophy – Junio Dort, PhD
Funded: 9/1/2021 - 8/31/2024
Grant Type:
Development Grant
Disease(s):
Duchenne muscular dystrophy (DMD)
Grantee:
Blocking RAN translation to rescue C9ORF72-related ALS/FTD phenotypes – Xin Jiang, PhD
Funded: 9/1/2021 - 8/31/2024
Grant Type:
Development Grant
Disease(s):
ALS (amyotrophic lateral sclerosis)
Grantee:
Role of mitochondrial hypoxia inducible factors in respiratory chain biogenesis – Alba Timón-Gómez, PhD
Funded: 9/1/2021 - 8/31/2024
Grant Type:
Development Grant
Disease(s):
Mitochondrial myopathies
Grantee:
Data Resource for CMT Genomics - Stephan Zuchner, MD, PhD
Funded: 4/1/2021 - 12/31/2023
Grant Type:
Infrastructure Grant
Disease(s):
Charcot-Marie-Tooth disease (CMT)