Rare Diseases

Rare diseases were once considered medical curiosities with little public-health impact. But though such diseases are individually rare, collectively an estimated 25 to 30 million Americans are affected. NIH’s Undiagnosed Disease Program focuses on the most puzzling medical cases referred to the NIH Clinical Center in Bethesda, Maryland. UDP has received nearly 10,000 inquiries, reviewed more than 3,000 applications, and admitted about 900 patients to the NIH Clinical Center for comprehensive weeklong evaluations. Some of these patients with rare diseases have taught us more about common conditions such as osteoporosis, kidney stones, and viral infections. Building on the early successes of the NIH UDP, NIH has extended the program into a network of sites across the country. Advances in diagnosis of rare diseases are gratifying, but are not enough: Of the 7,000 identified rare and neglected diseases for which we know the molecular cause, only about 500 have approved treatments. Through the Therapeutics for Rare and Neglected Diseases program of the National Center for Advancing Translational Sciences (NCATS) and other research efforts, NIH is collaborating with multiple partners to speed up the development of effective treatments.

02xl  is your source for accurate and trustworthy information so you can make the best choices for your health and wellness.

 

PS: Please note that 02xl provides this information for the benefit of the rare disease and overweight community. 02xl is not a medical provider or health care facility and thus can neither diagnose any disease or disorder nor endorse or recommend any specific medical treatments. Patients must rely on the personal and individualized medical advice of their qualified health care professionals before seeking any information related to their particular diagnosis, cure or treatment of a condition or disorder.

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