PAR-25-227
Natural History of Disorders Screenable in the Newborn Period (R01 Clinical Trial Optional)
Summary
PAR-25-227: Natural History of Disorders Screenable in the Newborn Period
Research Focus
This program funds research to characterize the natural history of disorders that are or may become part of statewide newborn screening (NBS) programs. The funder seeks longitudinal studies documenting the sequence, timing, and progression of symptoms in infants and children identified through screening, with emphasis on genotype-phenotype correlations, genetic and clinical heterogeneity, and modifying genetic, epigenetic, or environmental factors. Research should generate comprehensive data to identify underlying biological mechanisms, improve diagnostic accuracy, establish baselines for intervention trials, and support development of targeted, age-appropriate treatments. Priority conditions include inborn errors of metabolism, lysosomal storage disorders, hemoglobinopathies, congenital immunodeficiency syndromes, mitochondrial disorders, neuromuscular disorders (spinal muscular atrophy, muscular dystrophies), and genetic/epigenetic syndromes (Fragile X and others). Studies may also establish patient registries or longitudinal data collection systems to track child and family outcomes following newborn screening.
At a Glance
- Who can apply: Not stated
- Funding & project length: Not stated
- Award mechanism: R01 Research Project Grant (clinical trial optional)
- Key dates: Applications due February 5, 2025 (earliest); subsequent rounds June 5, 2025 and October 5, 2025; expiration January 8, 2028
- Best fit for: Pediatric researchers, geneticists, and metabolic disease specialists conducting prospective cohort studies or registry development in newborn screening populations
Key Facts
Deadline
Fri, January 7, 2028
Posted
Wed, November 6, 2024
Keywords
Research Areas
Gotchas (3)
The FOA states 'Number of Applications: See Section III. 3. Additional Information on Eligibility' but the provided text does not include Section III.3, making it impossible to determine if there are
90%
Source Text
“Number of Applications: See Section III. 3. Additional Information on Eligibility.”
This PAR was updated March 31, 2025 to align with agency priorities, but the document does not specify what changed from the prior version (PAR-21-115). Applicants may unknowingly submit applications
75%
Source Text
“This funding opportunity was updated to align with agency priorities. Carefully reread the full funding opportunity and make any needed adjustments to your application prior to submission.”
The FOA references Notice NOT-OD-24-084 regarding 'Overview of Grant Application and Review Changes for Due Dates on or after January 25, 2025' but does not summarize what those changes are. Applicant
75%
Source Text
“April 4, 2024 - Overview of Grant Application and Review Changes for Due Dates on or after January 25, 2025. See Notice NOT-OD-24-084.”