ArticleClinical drug investigation2023
Pharmacokinetics, Safety, and Tolerability of Single-Dose Orally Administered Venglustat in Healthy Chinese Volunteers.
Article in Clinical drug investigation, 2023. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 2 papers, 1 of them a synthesis that pooled it.
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The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
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2 citing papers in PubMed, 1 synthesis or guideline pooled it, 2 citations in OpenAlex.
- Fabry disease cardiomyopathy: A state-of-the-art review.Progress in cardiovascular diseasesPooled it
- Effectiveness and safety of enzyme replacement therapy in the treatment of Fabry disease: a Chinese monocentric real-world study.Orphanet journal of rare diseases · 2024Article
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7 authors at 2 institutions in 1 country.
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No grant is acknowledged in the PubMed record.
Abstract
BACKGROUND AND
objectiveDeficiencies of enzymes acting downstream of glucosylceramide synthase (GCS) can cause severe substrate accumulation. Venglustat is a small-molecule, brain-penetrant GCS inhibitor under investigation for multiple diseases involving pathogenic glycosphingolipid accumulation. Here, we evaluate the pharmacokinetics, safety, and tolerability of venglustat in healthy Chinese volunteers.
methodsStudy PKM16116 was a phase I, single-center, non-randomized, open-label study to investigate the pharmacokinetics, safety, and tolerability of a single 15 mg dose of orally administered venglustat in healthy Chinese volunteers aged 18 to 45 years.
resultsA total of 14 volunteers (7 male; 7 female) with a body mass index from 20.9 kg/m
conclusionVenglustat showed a favorable pharmacokinetic, safety, and tolerability profile in healthy Chinese volunteers following a single oral 15 mg dose. CLINICAL TRIAL REGISTRY NO: CTR20201012 ( http://www.chinadrugtrials.org.cn ) registered on 24 February 2021 and ChiCTR2200066559 ( http://www.chictr.org.cn ) retrospectively registered on 9 December 2022.
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