Marvel Biosciences’ lead experimental oral therapy MB-204 moved multiple measures of behavior and cognitive function toward normal levels in a mouse model of fragile X syndrome (FXS), with some signs of a potential carry-over effect after treatment was discontinued. “This is now the third ASD [autism spectrum disorder]…
Discussion
July is Fragile X Awareness Month, and patients, their families, advocates, researchers, and healthcare professionals are coming together — in a variety of efforts, including a Pokémon tournament — to increase understanding and spread knowledge of fragile X syndrome, a rare genetic condition that supporters say remains unfamiliar to…
Fragile X syndrome is a genetic condition associated with cognitive impairment, learning and behavioral challenges, and several physical features. Learn more about the disease here.
Fragile X is caused by mutations in the fragile X mental retardation 1 (FMR1) gene, located in the X chromosome. Learn more about what causes the disease here.
Symptoms differ in severity and occurrence among patients. Life expectancy for fragile X patients is generally normal. Learn more about the symptoms of the disease here.
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