KER-0193, an oral therapy being developed as a fragile X syndrome treatment, has been awarded orphan drug and rare pediatric disease designations by the U.S. Food and Drug Administration (FDA). These designations — which follow news of positive Phase 1 trial data from a test in healthy volunteers and…
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Long-term, daily treatment with the investigational cannabidiol (CBD) gel ZYN002 — also known as Zygel — led to clinically meaningful reductions in irritability and other behavioral symptoms in people with fragile X syndrome. That’s according to up to three years of data from an ongoing open-label extension…
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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