A first-of-its-kind organization launched on May 13 unifying the voices of life science companies committed to delivering desperately needed treatments to rare disease patients. The Rare Disease Company Coalition’s 10 founding members have brought 22 treatments to market and have more than 160 rare disease programs in the works.
Alnylam Pharmaceuticals reported positive results for the ILLUMINATE-B Phase III clinical trial evaluating the RNAi therapeutic lumasiran in young children with primary hyperoxaluria type 1 (PH1).
Alnylam’s gene-silencing therapy for a rare kidney disorder met the main goal of a late-stage study, bringing the company a step closer to marketing the first approved treatment for the condition.
