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Stock impact report

Ushering in a new era of fit-for-purpose clinical endpoints for Rett syndrome [Yahoo! Finance]

ACADIA Pharmaceuticals Inc. (ACAD) 
Last acadia pharmaceuticals inc. earnings: 2/26 04:05 pm Check Earnings Report
Company Research Source: Yahoo! Finance
As much of a trial's success depends on the ability of outcome measures to assess features most significant to the disease, experts say clinical studies for the rare disorder Rett syndrome need better measures to objectively confirm symptom improvement. Rett syndrome is a rare X-linked dominant disorder caused by a mutation in the MECP2 gene on the X chromosome. The condition, which is almost exclusively found in females, goes unnoticed for the first 6–18 months of life, and then slowly affects brain development, resulting in severe physical and mental impairments. In recent times, there have been clinical advances in the Rett syndrome therapeutics space. In March 2023, Acadia Pharmaceuticals'Daybue (trofinetide) became the first and only US FDA-approved treatment for Rett syndrome. However, in January 2024, another late-stage drug, Anavex Life Sciences' ANAVEX2-73 (blarcamesine), failed in a pivotal Phase III study, tanking the company's stock by more than 36%. With an endgame Show less Read more
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