Rare diseases Research Funding: NIH Grants

• 495NIH projects (fiscal year 2025)
• $347.6 millionawarded (fiscal year 2025)

Each number has its full source under its own list below.

1. 1Access For All In Als (all Als) West Clinical Coordinating CenterSt. Joseph's Hospital And Medical Center · fiscal year 2025$20.2 million
2. 2Access For All In Als (all Als) East Clinical Coordinating CenterMassachusetts General Hospital · fiscal year 2025$14.4 million
3. 3Intermediate-sized Expanded Access Protocol For Cnm-au8 In Amyotrophic Lateral Sclerosis (als).New York University School Of Medicine · fiscal year 2025$11.6 million
4. 4Pre-symptomatic Familial Als (pre-fals) Study: From Prodrome And Biomarkers To Als/ftd PreventionUniversity Of Miami School Of Medicine · fiscal year 2025$11.3 million
5. 5Scalable Expanded Access With Analysis Of Neurofilament And Other Biomarkers For Ibudilast In Als (sea-nobi-als)Mayo Clinic Jacksonville · fiscal year 2025$5.7 million
6. 6Data Management And Coordinating Center: Rare Diseases Clinical Research NetworkCincinnati Childrens Hosp Med Ctr · fiscal year 2025$5.6 million
7. 7Silence Als: A Platform For The Discovery And Development Of Antisense Therapeutics For Patients With Ultra-rare Forms Of AlsColumbia University Health Sciences · fiscal year 2025$4.6 million
8. 8Cmc Support For Ncats Rare Disease Platform Gene Therapy Trial ProjectsLeidos Biomedical Research, Inc. · fiscal year 2025$4.5 million
9. 9Advancing Rare Disease Discovery Through Technological And Methodological Approaches Using An Intuitive Platform, SeqrBroad Institute, Inc. · fiscal year 2025$3.3 million
10. 10Genetic And Clinical Studies Of Gaucher Disease And Other Lysosomal DisordersNational Human Genome Research Institute · fiscal year 2025$3.3 million

Source: NIH RePORTER, NIH-funded projects and award amounts, fiscal year 2025. Retrieved 12 Nov 2026.

• Genomics
• Brain and nerves
• Biologics and biosimilars
• Diagnostics and lab testing
• Clinical trials
• Cardiovascular
• Respiratory
• Pediatric
• Cell and gene therapy
• Autoimmune diseases