Checklist: Optimism In Action Scientists at Acadia Pharmaceuticals may soon offer new hope to sufferers of a mostly overlooked disorder.
Posted on Aug 21, 2019 12:10pm

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Having been granted Fast Track Status, as well as an Orphan Drug Designation, pharmaceutical innovator Acadia Pharmaceuticals will soon be initiating a Phase 3 trial—the one that determines FDA approval—evaluating the effectiveness of its proprietary Trofinetide in treating Rett syndrome.

Caused by a mutation in the X chromosome, Rett syndrome, a debilitating neurological disorder that occurs mostly in females, is often misdiagnosed as autism or cerebral palsy, and generally becomes evident somewhere between six and 18 months of age. Interfering with the brain activity responsible for cognitive, sensory, emotional, motor, and nervous-system function, its symptoms can also include scoliosis, sleep disturbances, disorganized breathing patterns, and seizures. There are no approved medications currently available to treat Rett syndrome, but with the randomized, placebo-controlled, double-blind study scheduled to begin in the fourth quarter of 2019, that could soon change.

Learn more about Rett Syndrome Research Trust, an organization on a mission to drive the development treatments and find a cure to reverse Rett.

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