Despite two recent clinical trial failures, Orphazyme A/S’s arimoclomol is still on track for its June 17 PDUFA date in treating Niemann-Pick disease type C. The newest stumble is in the pivotal study of arimoclomol for treating amyotrophic lateral sclerosis (ALS) as it failed to hit its primary and secondary endpoints.

Using the roundworm C. elegans as a "living test tube," researchers at the University of Florida have identified specific gut bacteria that promoted protein misfolding throughout the body, as well as others that were protective.

New genome editing techniques have opened up the number of potential mutations that could be addressed in the inherited muscle wasting disorder Duchenne muscular dystrophy, while also reducing the probability of inducing off target effects.