Project Grant R21TR006299
- This Project Grant award from the National Institute of Child Health and Human Development (NICHD), under the Child Health and Human Development Extramural Research program (CFDA 93.865), provides $178,000.00 to The Children's Hospital of Philadelphia (CHOP) to design and validate a Pelizaeus Merzbacher Disease (PMD) severity scale. The goal is to develop a clinician-reported outcome measure that can be used to stratify PMD patients by disease severity, particularly early in the disease...
- This Project Grant award from the National Institute of Neurological Disorders and Stroke (NINDS), under the Extramural Research Programs in the Neurosciences and Neurological Disorders (CFDA 93.853) program, provides $1,049,035.00 to The Children's Hospital of Philadelphia Research Institute to harmonize and curate the MLD-Core Project. This will create a clinical-trial ready natural history database using pooled data from 10 major leukodystrophy centers. The goal is to develop risk...
- This $152,761 federal Project Grant award from the Department of Defense's Military Medical Research and Development program (CFDA 12.420) supports a research study at The Children's Hospital of Philadelphia (CHOP) investigating the use of quantitative sodium magnetic resonance imaging (MRI) and chemical exchange saturation transfer (CEST) to analyze low-grade gliomas in children with neurofibromatosis type 1. The 2-year study, which commenced on September 15, 2023, aims to advance diagnostic...
- The federal Project Grant award of $249,999.00 was provided by the Defense Health Agency, under the Military Medical Research and Development program (CFDA 12.420), to the Children's Hospital Medical Center in Cincinnati, Ohio. The award, with a performance period from September 15, 2025 to September 14, 2027, supports the research project titled "Assessment of Smoldering Glial Activation in Pediatric-Onset MS by Novel Neuroimaging." This project aims to advance the medical...
- This $226,500 federal Project Grant award from the National Institute of Neurological Disorders and Stroke (NINDS), under the Extramural Research Programs in the Neurosciences and Neurological Disorders (CFDA 93.853) program, will support a 2-year research project to characterize and treat peripheral nervous system dysfunction in Sanfilippo syndrome (mucopolysaccharidosis type IIIA). The award will enable researchers at the University of Georgia Research Foundation to develop novel disease...
- This Project Grant award from the National Institute of Neurological Disorders and Stroke (NINDS), under the Extramural Research Programs in the Neurosciences and Neurological Disorders (CFDA 93.853) program, provides $742,352 to New York University School of Medicine to conduct the "Childhood Adversity Research Effort in Multiple Sclerosis (CARE.IN.MS)" study. The goal of this multi-year research project is to collaboratively develop and execute a comprehensive, culturally-informed...
- This $288,000 Project Grant was awarded on August 4, 2025 by the Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) under the Child Health and Human Development Extramural Research federal grant program (CFDA 93.865). The grant supports research conducted by the Van Andel Research Institute in Grand Rapids, Michigan to investigate fatty acid metabolism and identify potential therapeutic targets for treating mitochondrial fatty acid synthesis (MTFAS)...
- This $174,302 Project Grant awarded by the National Human Genome Research Institute (CFDA 93.172 - Human Genome Research) aims to characterize how adults with the childhood-onset genetic disease cystic fibrosis conceptualize their futures and plan their lives amid evolving prognoses and therapeutic innovations. The 5-year grant, awarded to the Trustees of the University of Pennsylvania, will use a disability studies framework to conduct qualitative interviews with adults with cystic fibrosis...
- This federal Project Grant award from the National Center for Advancing Translational Sciences (NCATS), CFDA 93.350, provides $489,500 to The Children's Hospital of Philadelphia (CHOP) to conduct research on the use of the compound Repsox for prophylactic intervention in Familial Platelet Disorder with associated Myeloid Malignancy (FPDMM) due to RUNX1 haploinsufficiency. The research aims to examine the effects of Repsox on RUNX1 expression, activity, and isoform usage in FPDMM hematopoietic...
- This $3,227,502 federal Project Grant award from the National Institute of Diabetes and Digestive and Kidney Diseases (CFDA 93.847) is funding research at the University of Pennsylvania to investigate the role of mitochondrial dysfunction in myelodysplastic syndrome (MDS) caused by mutations in the SRSF2 splicing factor. The research aims to elucidate how SRSF2 mutations disrupt oxidative phosphorylation and activate the PINK1-mediated mitophagy pathway to enhance MDS cell survival. The findings...
This federal Project Grant award of $489,500 from the National Center for Advancing Translational Sciences (NCATS) CFDA 93.350 program supports research to define patient-centric outcome measures for Multiple Sulfatase Deficiency (MSD), an ultra-rare neurodegenerative disorder. The project, led by The Children's Hospital of Philadelphia (CHOP), aims to (1) identify patient-centric tools for measuring the burden of MSD disease and defining clinical benefit, and (2) validate the use of disease-specific scales in MSD for potential use as clinical endpoints in future trials. This 2-year effort will capture the longitudinal performance of a panel of patient-reported and provider-administered outcome measures in 30 MSD subjects, creating a study platform to support future research and regulatory needs for this rare disease community.
Mod # | Description | ReasonForModification | Federal Obligation | Date |
|---|---|---|---|---|
| Not listed | $489.5k | 9/3/25 |