Mirum Pharmaceuticals, Inc. (Nasdaq: MIRM), a leading rare disease company, today announced that the first participant has been enrolled in the BLOOM Phase 2 clinical study evaluating MRM-3379 in ...

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 ...

An experimental drug appears to help people with a genetic disorder called Fragile X syndrome. Fragile X is the most common inherited cause of intellectual disabilities and autism. NPR's Jon Hamilton ...

Fragile X Syndrome: Symptoms, Diagnosis, Treatments and Complications: By Shreoshree Chakrabarty Fragile X Syndrome (FXS), also known as FX syndrome or fragile syndrome, is the most common inherited ...

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 ...

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.

Joel Richter receives funding from NIH and FRAXA. Sneha Shah receives funding from the FRAXA Research Foundation. Fragile X syndrome is a genetic disorder caused by a mutation in a gene that lies at ...

In a recent study posted to the bioRxiv preprint* server, an international team of researchers investigated interactions between severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2) ...

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 ...