For many people with fragile X, the mutated gene that causes symptoms is active rather than silenced. Thom Leach/Science Photo Library Fragile X syndrome is a genetic disorder caused by a mutation in ...

An antisense therapy restores production of the protein FMRP in cell samples taken from patients with fragile X syndrome. This breakthrough was possible because of the novel findings, also presented ...

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 ...

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 ...

An antisense therapy developed by Joel D. Richter, PhD, Sneha Shah, PhD, and Jonathan K. Watts, PhD, at UMass Chan Medical School and Elizabeth Berry-Kravis, MD, PhD, at RUSH University Medical Center ...

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, the most common form of inherited intellectual disability, may be unfolding in brain cells even before birth, despite typically going undiagnosed until age 3 or later. A new study ...

After its cannabidiol gel candidate failed a phase 3 trial in fragile X syndrome, Harmony Biosciences has now paused development of the asset in a different genetic disease. Harmony announced in ...

The cover artistically integrates multiple elements central to investigating the developmental origins of Fragile X syndrome. A vibrant DNA double helix structure rendered in rainbow iridescent colors ...