Andrea Lobo,  —

Articles by Andrea Lobo

JR-441 enzyme replacement therapy named orphan drug by FDA

An experimental enzyme replacement therapy (ERT), called JR-441, for Sanfilippo syndrome type A has been designated an orphan drug by the U.S. Food and Drug Administration, the treatment’s developer, JCR Pharmaceuticals, has announced. The designation is given to therapies being developed for conditions affecting fewer than 200,000 people…