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论文共 41 篇作者统计合作学者相似作者

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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,Heather Lau,Camilo Toro,Jean M. Johnston,Robert Krupnick,Alaa Hamed,Gerald F. Cox

MOLECULAR GENETICS AND METABOLISMno. 3 (2024)

引用1浏览0WOS引用

A Case Study of 2-Stage Seamless Adaptive Sample Size Re-Estimation Design with Efficacy Interim Analysis when Slope is the Primary Endpoint

Qi Zhang, Yuqian Shen,Hui Quan,Pascal Minini, Lin Wang

STATISTICS IN BIOPHARMACEUTICAL RESEARCHno. 2 (2024): 218-228

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Changes in Hematologic and Visceral Manifestations over Time Following Imiglucerase Initiation in Gaucher Disease Type 1 and Type 3 Pediatric Patients in the ICGG Gaucher Registry

Pramod K. Mistry,Pablo Bianculli,Isabela Batsu,Walter Heine, Antonio Oliveira-dos-Santos,Pascal Minini,Julie Batista

Molecular Genetics and Metabolismno. 2 (2023): 107225-107225

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Venglustat, a Novel Brain-Penetrant Glucosylceramide Synthase Inhibitor, for Gaucher Disease Type 3: Phase 3 LEAP2MONO Trial Design

Walter Heine,Isabela Batsu,Pascal Minini, Dmitry Cherkasov

Molecular Genetics and Metabolismno. 2 (2023): 107144-107144

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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,Guenter U. Hoeglinger,Kenneth Marek,Claudio Pacchetti,Anthony H. Schapira,Clemens R. Scherzer,Tanya Simuni,Pascal Minini,S. Pablo Sardi,M. Judith Peterschmitt

The Lancet Neurologyno. 8 (2023): 661-671

引用12浏览0WOS引用

Venglustat Combined with Imiglucerase for Neurological Disease in Adults with Gaucher Disease Type 3: the LEAP Trial

Raphael Schiffmann,Timothy M. Cox,Jean-Francois Dedieu,Sebastiaan J. M. Gaemers,Julia B. Hennermann,Hiroyuki Ida,Eugen Mengel,Pascal Minini,Pramod Mistry,Petra B. Musholt,David Scott,Jyoti Sharma,M. Judith Peterschmitt

BRAIN（2023）

引用15浏览0WOS引用

Two Years of Venglustat Combined with Imiglucerase Shows Continued Positive Effects on Neurological Features and Brain Connectivity in Adults with Gaucher Disease Type 3

Isabela Batsu,Riliang Zheng,Pascal Minini, Dmitry Cherkasov,David Scott,Ozlem Goker-Alpan,Julia B. Hennermann,Ken Sakurai,Timothy M. Cox

MOLECULAR GENETICS AND METABOLISMno. 2 (2023): 15-15

引用1浏览0引用

Fine-motor Functioning and Its Relevance to Activities of Daily Living in Adults with Late-Onset GM2 Gangliosidosis: Analysis of Baseline Data of AMETHIST Trial

Timothy M. Cox, Maria B. Rodriguez, Valerie Dyevre,Jerome Msihid,Chad Gwaltney,Ruth Pulikottil-Jacob,Riliang Zheng,Pascal Minini,Isabela Batsu

MOLECULAR GENETICS AND METABOLISMno. 2 (2023): 29-29

引用0浏览0引用

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,Shigeo Horie,Bertrand Knebelmann,Michal Mrug,Albert C. M. Ong,York P. C. Pei,Vicente E. Torres,Vijay Modur,Igor Antonshchuk,Ronald D. Perrone

American Journal of Kidney Diseases（2022）

引用6浏览0WOS引用

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