Diseases, Conditions, Syndromes

Research reveals new treatment strategy for propionic acidemia

Scientists at St. Jude Children's Research Hospital have shown that a class of drugs they pioneered has the potential to treat a rare metabolic disorder called propionic acidemia. The findings were published today in Science ...


Gene therapy for rare form of blindness wins US approval

U.S. health officials on Tuesday approved the nation's first gene therapy for an inherited disease, a treatment that improves the sight of patients with a rare form of blindness. It marks another major advance for the emerging ...


New biomarker found for group of rare metabolic diseases

A newly discovered biomarker associated with a rare metabolic disorder may facilitate better diagnosis and identification of new drugs for clinical trials for the disease, according to researchers in the Perelman School of ...


FDA drug approvals reached 18-year high in 2014

The Food and Drug Administration approved 41 first-of-a-kind drugs in 2014, including a record number of medicines for rare diseases, pushing the agency's annual tally of drug approvals to its highest level in 18 years.

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