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, ...

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, ...

Building on more than two decades of research, a study by MIT neuroscientists at The Picower Institute for Learning and ...

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 ...

A new study shows that enhancing activity of a specific component of 'NMDA' receptors normalizes protein synthesis, neural activity and seizure susceptibility in the hippocampus of fragile X lab mice.

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 ...