Rare diseases affect fewer than 200,000 persons and in the States of America, but, with more than 7,000 rare diseases having been identified, millions of Americans actually suffer from them.
Correct diagnosis and treatment require the specialty expertise and infrastructure common to great medical centers.
Here are the top 10 hospitals in the USA recognized for their advanced research, clinical trials, and specialized care for rare diseases in 2025:
1. National Institutes of Health (NIH) Clinical Center – Bethesda, Maryland
- Why it takes the number one spot: It is the largest hospital in the world dedicated to clinical research strictly and has dozens of programs for ultra-rare diseases and undiagnosed diseases.
- Highlight: It is home to the Undiagnosed Diseases Program (UDP) and various NIH-funded studies on rare genetic disorders.
2. Mayo Clinic – Rochester, Minnesota
- Reason for being top-tier: The Mayo Clinic beefs itself up by having one of the most exigent diagnostic laboratory setups in the world to front the identification and treatment of rare autoimmune, neurological or genetic disorders.
- Highlights: This institute is reputed for immunizing against medical mysteries and for working at the forefront of individualized medicine.
3. Cleveland Clinic – Cleveland, Ohio
- Speciality: Multisystem rare diseases, neurological disorders, and rare cancers.
- For what it stands out: Presenting interdisciplinary teams addressing complex multisystem diseases, such as Ehlers-Danlos syndrome, amyloidosis, and rare cardiac conditions.
- Spotlight: Center for Rare Diseases and team-based care approach.
4. Massachusetts General Hospital – Boston, Massachusetts
- Affiliated With: Harvard Medical School
- Why it’s recognized: Is well noted for particular programs in rare blood disorders, mitochondrial diseases, and rare cancers.
- The Highlight: Center for Genomic Medicine and frequent clinical trials in many areas of front-line rare diseases.
5. Stanford Health Care – Stanford, California
- Specialty: Genetic and neurological rare diseases
- Why it is highly ranked: Stanford offers hope to families suffering from disorders such as Rett syndrome, rare epilepsies, and rare inherited disorders by coupling genomic research with patient care.
- Highlight: Stanford Center for Undiagnosed Diseases.
6. Johns Hopkins Hospital – Baltimore, Maryland
- The reason for its top rating: Famous for its leadership in arriving at research and care for rare autoimmune, metabolic, and infectious diseases.
- Highlight: Home to the Greenberg Center for Skeletal Dysplasias and the Johns Hopkins Myositis Center.
7. University of California, San Francisco (UCSF) Medical Center – San Francisco, California
- Why it is so named: UCSF stands a head and shoulders above the rest in the care of pediatric and adult rare diseases, especially metabolic and mitochondrial disorders.
- Highlight: Institute for Human Genetics program and rare pediatric programs at UCSF.
8. Children’s Hospital of Philadelphia (CHOP) – Philadelphia, Pennsylvania
- Field: Pediatric rare diseases
- What makes it exceptional: CHOP is one of the global leaders in the rare pediatric condition treatment and is a renowned site of many gene therapy clinical trials.
- Highlight: Raymond G. Perelman Center for Cellular and Molecular Therapeutics for rare genetic disorders.
9. Mount Sinai Hospital – New York, New York
- The reason it makes the list: Whenever a person has lysosomal storage disorders, rare endocrine disorders, or rare cancers, there is expert care offered at Mount Sinai.
- Highlight: The Icahn Institute for Genomics and Multiscale Biology acts as a hub for rare disease discovery.
10. Duke University Hospital – Durham, North Carolina
- Field: Rare genetic and neurological diseases
- Why it is trusted: Duke is known for its Center for Human Disease Modeling and its work in gene therapy and rare neurodevelopmental disorders.
- Highlight: Comprehensive rare disease registry and access to experimental treatments.