CRADA combines Scenic Biotech's genetic modifier platform with NIH's patient registry to unlock genetic modifiers as a path to novel therapies for Niemann-Pick Type C disease Scenic Biotech, a pioneer
Riluzole, a drug approved to treat amyotrophic lateral sclerosis (ALS), a disease affecting nerve cells controlling movement, could slow the gradual loss of a particular brain cell that occurs in Niemann-Pick disease type C1 (NPC1), a rare genetic disorder affecting children and adolescents, suggests a study in mice by scientists at the National Institutes of Health.
Repurposed ALS drug shows promise in mouse model of rare childhood genetic disorder miragenews.com - get the latest breaking news, showbiz & celebrity photos, sport news & rumours, viral videos and top stories from miragenews.com Daily Mail and Mail on Sunday newspapers.