Spinogenix’s Fragile X Syndrome (FXS) drug is the first to show rescued well-established resting EEG abnormalities, ...

Spinogenix, Inc., a clinical-stage biopharmaceutical company pioneering first-in-class therapeutics that restore synapses to improve the lives of patients worldwide, today announced the publication of ...

In a comprehensive Genomic Press Interview, researchers from the University of Texas Health Science Center at San Antonio and Hirosaki University have uncovered critical new insights into the ...

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 ...

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 ...

Les Laboratoires Servier said that it agreed to acquire a potential treatment for Fragile X syndrome (FXS), the most common genetic cause of autism spectrum disorder (ASD), from its original developer ...

Enthorin Therapeutics Announces Initiation of Phase II Clinical Trial of MRM-3379 (Formerly ENT-3379) for Fragile X Syndrome by Licensing Partner Mirum Pharmaceuticals Enthorin Therapeutics, LLC, a ...

A new study suggests a potential molecular strategy for treating fragile X syndrome, an inherited neurodevelopmental disorder that causes autism spectrum disorder and intellectual disability. This ...

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 ...

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.

For 22 years, Jason Mazzola’s life was defined by Fragile X, a genetic condition that often causes autism and intellectual disability. Jason, who is 24 now, needed constant supervision. He had ...