Tag Archives: rare

FDA grants Jacobus Pharma approval for rare disease drug

(Reuters) – Jacobus Pharmaceutical Co Inc on Monday won U.S. approval for the first drug to treat children with Lambert-Eaton myasthenic syndrome, a rare autoimmune disorder. The drug, Ruzurgi, was approved for use in patients aged between 6 and 17, the FDA said here. Lambert-Eaton myasthenic syndrome, which affects about three people per million worldwide,… Read More »

First US attempt to cure a rare disease with genome editing fails miserably 

First US attempt to cure a rare disease with genome editing fails miserably Scientists at Sangamo attempted the first human trial of a gene-editing method to cure rare, debilitating enzyme deficiency diseases  After showing early promise, the DNA replacement failed to reduce levels of harmful unprocessed sugars  The gene replacement itself worked, but after nearly… Read More »