GOV — GOVERNMENT

Clinical Trial Readiness for Rare Diseases (R21)

NCATS + NICHD / Office of Rare Diseases Research (PAR-25-450)

Award

$275K direct total over 2 years

Deadline

Next due Feb 17, 2027; expires Jul 2028

Award type

Cash grant

Focus

Registries & biomarkers · Clinical research

Funds the development and validation of biomarkers, clinical outcome assessments, and natural-history data needed before a rare-disease trial can start.

Why it's in this index

BTHS-applicable endpoints include cardiac function measures, functional outcomes, and the monolysocardiolipin/cardiolipin ratio as a biomarker.

Who can apply

US institutions of all types; foreign organizations ineligible. Applicants need clinical expertise in the disease and access to sufficient patients; BSF's registry and clinics are relevant resources.

How to apply

Via eRA Commons / Grants.gov / ASSIST under PAR-25-450; Feb/Jun cycles through 2028, next due date Feb 17, 2027.

Links & downloads

PAR-25-450 ↗