Publications by authors named "P Minini"

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Venglustat in GM2 gangliosidoses and related disorders: Results of the AMETHIST randomized controlled and basket trials.
Cynthia J Tifft, Isabela Batsu, Roberto Giugliani, Harmonie Goyeau, Andreas Hahn, Pascal Minini

Genet Med· January 2026

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Clinical outcome assessments of disease burden and progression in late-onset GM2 gangliosidoses.
Julie Kissell, Camille Rochmann, Pascal Minini, Florian Eichler, Christopher D Stephen

Mol Genet Metab· July 2024

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Safety and efficacy of venglustat in GBA1-associated Parkinson's disease: an international, multicentre, double-blind, randomised, placebo-controlled, phase 2 trial.
Nir Giladi, Roy N Alcalay, Gary Cutter, Thomas Gasser, Tanya Gurevich, Pascal Minini

Lancet Neurol· August 2023

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Venglustat, a Novel Glucosylceramide Synthase Inhibitor, in Patients at Risk of Rapidly Progressing ADPKD: Primary Results of a Double-Blind, Placebo-Controlled, Phase 2/3 Randomized Clinical Trial.
Ronald T Gansevoort, Ali Hariri, Pascal Minini, Curie Ahn, Arlene B Chapman

Am J Kidney Dis· May 2023

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Venglustat combined with imiglucerase for neurological disease in adults with Gaucher disease type 3: the LEAP trial.
Raphael Schiffmann, Timothy M Cox, Jean-François Dedieu, Sebastiaan J M Gaemers, Julia B Hennermann, Pascal Minini

Brain· February 2023

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