An orphan drug designation for a stem cell therapy (Prochymal; Osiris Therapeutics Inc.) in patients with newly diagnosed type 1 diabetes mellitus has recently been approved by the U.S. Food and Drug Administration. Related terms:type 1 diabetes n orphan diseasetype 1 diabetes orphanOsiris and Type1 Diabetes andorphan disease diabetes type iiOsiris and Type1 Diabetes and […]

Xenazine (tetrabenazine) has been approved by the United States Food and Drug Administration (US FDA) as the first drug for the treatment of chorea among patients with Huntington’s disease. “Xenazine represents hope for patients and families dealing with this difficult disease,” said Dr. Timothy Cote, director of FDA’s Office of Orphan Products Development. “For the […]