Servier is souping up its neurology pipeline with a clinical-stage candidate sourced from U.K. biotech Kaerus Bioscience for a rare genetic disease. The French pharma is offering as much as $450 ...

Mutations in FMR1, the gene encoding fragile X messenger ribonucleoprotein 1 (FMRP), located at Xq27.3 and discovered in 1991, are the leading single-gene causes of autism and intellectual disability.

Fragile X Syndrome: Symptoms, Diagnosis, Treatments and Complications: By Shreoshree Chakrabarty Fragile X Syndrome (FXS), also known as FX syndrome or fragile syndrome, is the most common inherited ...

Data to be Presented at the Hot Topics in Child Neurology Podium Presentation Session at the American Academy of Neurology Annual Meeting PLYMOUTH MEETING, Pa.--(BUSINESS WIRE)-- Harmony Biosciences ...

Proof-of-concept study underway for investigational oral therapy in Fragile X syndrome; top-line data from Phase 2 expected in 2027 Mirum Pharmaceuticals, Inc. (Nasdaq: MIRM), a leading rare disease ...

DEVON, Pa., May 02, 2022 (GLOBE NEWSWIRE) -- Zynerba Pharmaceuticals, Inc. (Nasdaq: ZYNE), the leader in innovative pharmaceutically-produced transdermal cannabinoid therapies for rare and near-rare ...