Zevra therapeutics presented new data for arimoclomol and olpruva® (sodium phenylbutyrate) at the society for the study of inborn errors of metabolism (ssiem) 2024 annual symposium

New clinical efficacy and safety data for arimoclomol as a possible treatment for niemann-pick disease type c, including from long-term and real-world settings, demonstrate clinically meaningful reduction in disease progression
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