Project Grant K23NS144702
- The National Institute of Neurological Disorders and Stroke awarded Virginia Commonwealth University $194,907 on July 10, 2026, under the Extramural Research Programs in the Neurosciences and Neurological Disorders program (CFDA 93.853) to support a mentored career development project identifying genetic modifiers of disease severity in myotonic dystrophy type 1. Dr. Samuel Carrell, an Assistant Professor of Neurology at Virginia Commonwealth University, leads the research effort. The award...
- The National Institute of Arthritis and Musculoskeletal and Skin Diseases awarded Virginia Commonwealth University $2,776,749 on September 25, 2025, under the Arthritis, Musculoskeletal and Skin Diseases Research program (CFDA 93.846) to conduct research on myotonic dystrophy type 1 (DM1) across pediatric and adult populations. The award funds two research projects designed to close gaps in clinical trial readiness for emerging DM1 therapeutics. Project 1 enrolls 150 children with congenital...
- The National Institute of Neurological Disorders and Stroke awarded Regenerative Research Foundation $233,550 on June 1, 2026, under the Extramural Research Programs in the Neurosciences and Neurological Disorders program (CFDA 93.853) to model myotonic dystrophy type 1 and type 2 neuropathology using human induced pluripotent stem cell-derived cortical organoids. The project generates disease and control cortical organoids from DM1 and DM2 patient-derived iPSC lines and healthy controls using...
- The National Institute of Neurological Disorders and Stroke (NINDS), a component of the Department of Health and Human Services National Institutes of Health, awarded Baylor College of Medicine $124,106 on April 1, 2026, under the Extramural Research Programs in the Neurosciences and Neurological Disorders program (CFDA 93.853, Project Grant K99NS146618). The award funds research into the mechanisms of neurological manifestations in myotonic dystrophy type 1 (DM1), a progressive neuromuscular...
- The National Institute of Child Health and Human Development awarded Washington University $667,506 on September 1, 2026, under the Child Health and Human Development Extramural Research program (CFDA 93.865) to develop a caregiver-reported dystonia impact scale for children with cerebral palsy. The project addresses underdiagnosis of dystonia in cerebral palsy, a condition affecting 1 in 500 people in the United States. Dystonia commonly causes motor functional limitations in childhood but is...
- The National Institute of Neurological Disorders and Stroke awarded the University of Virginia $490,593 on September 1, 2026, under the Extramural Research Programs in the Neurosciences and Neurological Disorders program (CFDA 93.853) to investigate the role of growth differentiation factor 11 (GDF11) in neurodevelopmental disorders. The research examines GDF11 as a molecular hub involved in abnormal brain function across multiple genetically distinct neurodevelopmental disorders. The first...
- Federal Grant Award Summary The National Institute of Neurological Disorders and Stroke (NINDS) awarded a $519,166 Project Grant on August 8, 2025, to the Research Institute at Nationwide Children's Hospital to support preclinical development of a vectorized exon skipping (VES) therapeutic approach for Duchenne Muscular Dystrophy (DMD) exon 44 mutations. Under the Extramural Research Programs in the Neurosciences and Neurological Disorders program (CFDA 93.853), the awardee will develop and...
- The National Institute of Neurological Disorders and Stroke awarded $616,577 to the Research Institute At Nationwide Children's Hospital on August 14, 2026, under the Extramural Research Programs in the Neurosciences and Neurological Disorders program (CFDA 93.853) to elucidate cellular and molecular disease mechanisms in Charcot-Marie-Tooth type 1B disease and develop gene therapy approaches. The recipient will pursue two aims. In Aim 1, the institute will determine how myelin protein zero...
- The National Institute of Neurological Disorders and Stroke awarded the University of Missouri System $574,083 on March 15, 2026, under the Extramural Research Programs in the Neurosciences and Neurological Disorders program (CFDA 93.853) to develop a vector-based therapeutic for Charcot-Marie-Tooth disease type 2E (CMT2E) using a novel mouse model. The recipient will develop a precision medicine approach to treating CMT2E, which is caused by mutations in the neurofilament light gene (NEFL). The...
- The National Institute of Neurological Disorders and Stroke awarded Albany Medical College $164,000 on August 10, 2026, under the Extramural Research Programs in the Neurosciences and Neurological Disorders program (CFDA 93.853) to explore the pathophysiology of KCTD11-related Charcot-Marie-Tooth neuropathy. The project investigates how mutations in KCTD11 cause recessive intermediate CMT (RI-CMTE), a peripheral neuropathy characterized by progressive neurological decline and abnormal...
The National Institute of Neurological Disorders and Stroke awarded Virginia Commonwealth University $173,335 on August 15, 2026, under the Extramural Research Programs in the Neurosciences and Neurological Disorders program (CFDA 93.853) to develop and validate clinical outcome assessments for congenital myotonic dystrophy in children. The project addresses gaps in measuring motor function change in congenital myotonic dystrophy (CDM), a severe inherited disorder characterized by progressive impairments in motor, cognitive, and cardiac function. CDM presents with severe weakness at birth, respiratory failure requiring ventilatory support, and feeding difficulties. Children who survive the initial critical illness show spontaneous improvement through early childhood followed by functional decline during adolescence and adulthood. Recent therapeutic trials targeting disease mechanisms in myotonic dystrophy have shown strength and function improvements in adults, but the lack of responsive clinical outcome measures has created a barrier to implementing therapeutic trials for children with CDM. Current clinical assessments exclude children under six years of age and do not adequately capture community-level motor function. The recipient will validate a gross motor function assessment for non-ambulatory children under age six and develop an objective measure of community-level motor function. These instruments will enable detection of treatment response in pediatric CDM populations and support inclusion of younger children in active therapeutic trials. The period of performance runs from award date through July 31, 2031. Work is performed in Richmond, Virginia.
Mod # | Description | ReasonForModification | Federal Obligation | Date |
|---|---|---|---|---|
| Not listed | $173.3k | 8/7/26 |