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 ...

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.

Building on more than two decades of research, a study by MIT neuroscientists at The Picower Institute for Learning and Memory reports a new way to treat pathology and symptoms of fragile X syndrome, ...

Until recently, scientists thought they understood one of the underlying causes of fragile X syndrome, the most common inherited cause of intellectual disability in the United States. The syndrome, ...

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

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 ...

Still, like many parents and caregivers of individuals with Fragile X — the most common inherited form of autism and it is one of the most common inherited causes of developmental and intellectual ...

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 ...

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 ...

Observations of the small protrusions that line the dendrites of neurons, called spines, were critical in understanding the function of NMDA receptors in the new study, as well as a precursor to the ...

Please provide your email address to receive an email when new articles are posted on . The FDA has granted fast-track designation to an investigational small molecule to treat individuals with ...

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 ...