Project Grant R03NS151642
- The National Institute of Neurological Disorders and Stroke awarded Albany Medical College $562,051 on March 6, 2026, under the Extramural Research Programs in the Neurosciences and Neurological Disorders program (CFDA 93.853) to investigate modulation of the Hippo pathway as a therapeutic approach for Charcot-Marie-Tooth disease type 1A (CMT1A). The research targets the YAP/TAZ-TEAD1 transcriptional complex, which regulates PMP22 expression in Schwann cells. CMT1A, the most common inherited...
- 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 Illinois $400,826 on June 1, 2026, under the Extramural Research Programs in the Neurosciences and Neurological Disorders program (CFDA 93.853) to support discovery-based research into the mechanisms of axonopathy in CMT1X, a Charcot-Marie-Tooth disease caused by mutations in the gene encoding connexin 32 (CX32). The research integrates analysis of Schwann cells, other peripheral nerve cells, and spinal motor...
- 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 The University of Texas Southwestern Medical Center $249,000 on February 15, 2026, under the Extramural Research Programs in the Neurosciences and Neurological Disorders program (CFDA 93.853, Assistance Listing R21NS146806) to conduct gene replacement therapy research for Charcot-Marie-Tooth neuropathies caused by GDAP1 mutations. The project develops and evaluates AAV9-mediated GDAP1 gene replacement therapy in newly...
- 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...
- 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 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 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 Neurological Disorders and Stroke, part of the National Institutes of Health, awarded Trustees of Tufts College (doing business as Tufts University School of Medicine) $453,750 on September 1, 2026, under the Extramural Research Programs in the Neurosciences and Neurological Disorders program (CFDA 93.853). The project will define the therapeutic potential of GSK3 inhibition in correcting molecular phenotypes of CTNNB1 syndrome patient-derived glutamatergic neurons....
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 myelination. CMT is the most common inherited peripheral neuropathy, affecting approximately 1 in 2,500 individuals, yet no disease-modifying therapies exist. The work will generate patient-relevant experimental models combining genetic, cellular, and molecular approaches to determine whether KCTD11 dysfunction primarily arises from Schwann cell dysfunction, motor neuron vulnerability, or both. The research will investigate how KCTD11 deficiency compromises myelin integrity and axon-glial communication, clarify the cellular origins of RI-CMTE, and define pathways destabilized by KCTD11 mutations. The proposed work will deliver the first mechanistic framework for KCTD11-related neuropathy and provide a community resource for studying this newly recognized disease subtype. The award period runs from August 10, 2026, through July 31, 2028, with performance at Albany Medical College in Albany, New York.
Mod # | Description | ReasonForModification | Federal Obligation | Date |
|---|---|---|---|---|
| Not listed | $164.0k | 8/7/26 |