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Bookmark or Share- Home - Genetic and Rare Diseases Information CenterIn fact, rare diseases are estimated to affect more than 30 million people in the United States. Each disease may be rare individually, but people with rare diseases often face similar challenges. These challenges may include accessing information, getting a diagnosis, and finding resources.
- List of Rare Diseases | A-Z Database | NORDWhat Is a Rare Disease? A rare disorder is a disease or condition that affects fewer than 200,000 Americans. Cumulatively, there are more than 10,000 rare diseases affecting more than 30 million Americans. NORD is committed to the identification, treatment, and cure of rare diseases through education, advocacy, research, and service programs.
- National Organization for Rare Disorders | NORDFind your rare disease community. For Organizations. For Clinicians & Researchers. NORD is dedicated to supporting education, elevating care, advancing research, and driving policy for rare diseases.
- Diseases - Genetic and Rare Diseases Information CenterExplore GARD's list of rare diseases. Filter by category or search by disease name, acronym, or synonym. Rare diseases found on GARD should not be used as policy statements of NCATS or the NIH.
- Rare Diseases | National Institutes of Health (NIH)Looking Forward. The Promise of Precision Medicine. Rare Diseases. Pompe disease is a rare, inherited disorder characterized by the deficiency of an enzyme called acid alpha-glucosidase (GAA). One of the primary features of Pompe disease is the progressive break down in communications between nerve and muscle cells.
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