FRAXA's early preclinical support helped establish the calcium-activated potassium ("BK") channel modulator mechanism behind SPG601. FRAXA also supported the development of another BK channel ...

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.

Researchers have developed an innovative new nanopore sequencing assay to identify carriers of fragile X syndrome (FXS), the leading cause of monogenic autism spectrum disorder and inherited ...

Numerous potential treatments for neurological conditions, including autism spectrum disorders, have worked well in mice but then disappointed in ...

PLYMOUTH MEETING, Pa.--(BUSINESS WIRE)--Harmony Biosciences Holdings, Inc. (Nasdaq: HRMY), today announced topline results from its Phase 3 registrational clinical trial (the RECONNECT Study) of ...

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 ...

Harmony Biosciences Holdings, Inc. is downgraded from Buy to Hold after a failed Phase 3 trial for ZYN002 in Fragile X syndrome. HRMY's pipeline setback and sNDA rejection raise concerns about ...

Fragile X disorders include two distinct conditions: fragile X syndrome (the most common single-gene cause of developmental delay and autism) and an entirely separate group of premutation syndromes.