In a first, Alnylam reports success silencing genes in children

Alnylam Pharmaceuticals set the stage for another drug approval after reporting Wednesday that it successfully treated children with a rare liver and kidney disease.   Alnylam’s (Nasdaq: ALNY) latest drug candidate targets a rare disorder called primary hyperoxaluria in which a substance called oxalate can build up and cause bladder stones, kidney failure and other issues. The drug, known as lumasiran, successfully reduced that buildup in a late-stage trial of 18 children under the age of six,…
Source: bizjournals.com Health Care:Pharmaceuticals headlines - Category: Pharmaceuticals Authors: Source Type: news