Servier acquires KER-0193, a potential treatment for Fragile X syndrome, developed by Kaerus Bioscience, a Medicxi company KER-0193, a novel, orally bioavailable small molecule, was granted Orphan ...

Research on fragile X syndrome, the most common inherited cause of mental retardation, has focused mostly on how the genetic defect alters the functioning of neurons in the brain. A new study focusing ...

Scientists have created a new therapeutic approach for testing fragile X syndrome, which is the most common genetic cause of autism spectrum disorder (ASD). This method aims to alter a type of neural ...

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.

Collaboration with UMass Chan Medical School confirmed FMR1-217 as well as an RNA-targeted mechanism to restore functional FMRP protein to develop potential treatments for FXS QurAlis’ preliminary ...

Philadelphia, June 12, 2025 – Researchers have developed an innovative new nanopore sequencing assay to identify carriers of fragile X syndrome (FXS), the leading cause of monogenic autism spectrum ...

Harmony Biosciences Holdings, Inc. (NASDAQ:HRMY) released topline results from its Phase 3 registrational clinical trial (the RECONNECT Study) of ZYN002 in Fragile X syndrome (FXS). Fragile X syndrome ...

PLYMOUTH MEETING, Pa.--(BUSINESS WIRE)-- Harmony Biosciences Holdings, Inc. (HRMY), today announced topline results from its Phase 3 registrational clinical trial (the RECONNECT Study) of ZYN002 in ...

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 ...

(RTTNews) - Harmony Biosciences Holdings, Inc. (HRMY), Tuesday announced the updated data from its Open-Label Extension or OLE study ZYN2-CL-017, evaluating the safety and effectiveness of ZYN002 in ...