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Zevra Therapeutics to Present Data Demonstrating Relevance of Swallow Domain in Niemann-Pick Disease Type C Clinical Severity Scale at 53rd Child Neurology Society Annual Meeting
Zevra Therapeutics (NASDAQ: ZVRA) announced the presentation of data validating the swallow domain's significance in the Niemann-Pick Disease Type C Clinical Severity Scale(NPCCSS) at the 53rd Child Neurology Society Annual Meeting. The study, presented by Dr. Elizabeth Berry-Kravis, involved 12 NPC and swallow experts evaluating scoring categories and instructions.
The findings confirmed that NPCCSS swallow response categories can be consistently interpreted, with score changes reflecting actual changes in patient swallow function. The revised scoring algorithm improves linearity by re-ranking dysphagia categories and creating a separate category for tube-feeding. The R4DNPCCSS endpoint, which includes this revised swallow domain along with ambulation, fine motor skills, and speech domains, showed a favorable treatment difference of -1.51 (P=0.0413) for arimoclomol compared to placebo in the Phase 2/3 study.
The findings confirmed that NPCCSS swallow response categories can be consistently interpreted, with score changes reflecting actual changes in patient swallow function. The revised scoring algorithm improves linearity by re-ranking dysphagia categories and creating a separate category for tube-feeding. The R4DNPCCSS endpoint, which includes this revised swallow domain along with ambulation, fine motor skills, and speech domains, showed a favorable treatment difference of -1.51 (P=0.0413) for arimoclomol compared to placebo in the Phase 2/3 study.
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