Zynerba Pharmaceuticals’ experimental cannabis-based gel Zygel was not statistically significant in improving aberrant behavior when compared to a placebo in patients with a neurological disorder called Fragile X syndrome.
San Diego-based Engrail Therapeutics launched with $32 million in a Series A financing round that was solely funded by Nan Fung Life Sciences (NFLS).
A review of some recently published scientific studies includes one for antihistamines and similar compounds that might slow down Huntington’s disease.
Cambridge, Massachusetts-based Acceleron Pharma announced that the company’s Phase II clinical trial of ACE-083 in patients with Charcot-Marie-Tooth disease (CMT) failed to show functional improvement.
Acadia Pharmaceuticals Inc. and Neuren Pharmaceuticals Limited announced that the U.S. Food and Drug Administration granted Rare Pediatric Disease designation to trofinetide for the treatment of Rett syndrome, a serious and rare neurological disorder.
Biogen and Sangamo Therapeutics announced a broad global licensing collaboration deal to develop and commercialize several compounds for a range of neurological and neuromuscular diseases.
An investigational medicine in late-stage development by Jazz Pharmaceuticals hit the mark in treating cataplexy and excessive daytime sleepiness (EDS) in adults with narcolepsy.
PTC Therapeutics Inc. announced an agreement to acquire Agilis Biotherapeutics Inc., a biotechnology company advancing an innovative gene therapy platform for rare monogenic diseases that affect the central nervous system.
