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Zevra Therapeutics Presents New Data on the Long-Term Safety and Efficacy of Arimoclomol as a Treatment for Niemann-Pick Disease Type C at the SIMD 45th Annual Meeting [Yahoo! Finance]

Zevra Therapeutics, Inc. (ZVRA) 
Company Research Source: Yahoo! Finance
CELEBRATION, Fla., April 15, 2024 (GLOBE NEWSWIRE) -- Zevra Therapeutics, Inc. (NasdaqGS: ZVRA) (Zevra, or the Company), a rare disease therapeutics company, today announced that new long-term real-world data from the arimoclomol expanded access program (EAP: NCT04316637 ) for the treatment of Niemann-Pick disease type C (NPC) was featured in an oral presentation during the Society for Inherited Metabolic Disorders (SIMD) 45 th Annual Meeting taking place at the Sheraton/Le Meridien Charlotte Hotel Complex in Charlotte, NC, from April 14-17, 2024. “These real-world data collected in the arimoclomol US EAP represent the first evidence to support the use of arimoclomol in adults with NPC, demonstrating a clinically meaningful slowing of disease progression,” noted Kristina Julich, M.D., Assistant Professor, Department of Neurology, at The University of Texas at Austin, Dell Medical School. “NPC presents a therapeutic challenge, lacking approved treatment options in the U.S. and invar Show less Read more
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