E-LIB Suche - Ergebnisse für: Minini, Pascal

1

Clinical outcome assessments of disease burden and progress..:

Kissell, Julie ; Rochmann, Camille ; Minini, Pascal...
Molecular Genetics and Metabolism. 142 (2024) 3 - p. 108512 , 2024

Link: https://doi.org/10.1016/..

2

Venglustat in GBA1-related Parkinson's disease – Authors' r..:

Sardi, S Pablo ; Giladi, Nir ; Alcalay, Roy N...
The Lancet Neurology. 23 (2024) 2 - p. 137-138 , 2024

Link: https://doi.org/10.1016/..

3

Venglustat, a novel brain-penetrant glucosylceramide syntha..:

Heine, Walter ; Batsu, Isabela ; Minini, Pascal.
Molecular Genetics and Metabolism. 138 (2023) 2 - p. 107144 , 2023

Link: https://doi.org/10.1016/..

4

A Case Study of 2-Stage Seamless Adaptive Sample Size Re-Es..:

Zhang, Qi ; Shen, Yuqian ; Quan, Hui..
Statistics in Biopharmaceutical Research. , 2023

Link: https://doi.org/10.1080/..

5

Two years of venglustat combined with imiglucerase shows co..:

Batsu, Isabela ; Zheng, Riliang ; Minini, Pascal...
Molecular Genetics and Metabolism. 138 (2023) 2 - p. 107024 , 2023

Link: https://doi.org/10.1016/..

6

Venglustat, a Novel Glucosylceramide Synthase Inhibitor, in..:

Gansevoort, Ronald T. ; Hariri, Ali ; Minini, Pascal...
American Journal of Kidney Diseases. 81 (2023) 5 - p. 517-527.e1 , 2023

Link: https://doi.org/10.1053/..

7

Fine-motor functioning and its relevance to activities of d..:

Cox, Timothy M. ; Rodriguez, Maria B. ; Dyevre, Valérie...
Molecular Genetics and Metabolism. 138 (2023) 2 - p. 107063 , 2023

Link: https://doi.org/10.1016/..

8

AMETHIST: Baseline characteristics from a phase 3 trial of ..:

Tifft, Cynthia J. ; Batsu, Isabela ; Zheng, Riliang.
Molecular Genetics and Metabolism. 138 (2023) 2 - p. 107335 , 2023

Link: https://doi.org/10.1016/..

9

Changes in hematologic and visceral manifestations over tim..:

Mistry, Pramod K. ; Bianculli, Pablo ; Batsu, Isabela...
Molecular Genetics and Metabolism. 138 (2023) 2 - p. 107225 , 2023

Link: https://doi.org/10.1016/..

10

The STAGED-PKD 2-Stage Adaptive Study With a Patient Enrich..:

Perrone, Ronald D. ; Hariri, Ali ; Minini, Pascal...
Kidney Medicine. 4 (2022) 10 - p. 100538 , 2022

Link: https://doi.org/10.1016/..

11

Venglustat, a novel brain-penetrant glucosylceramide syntha..:

Petrović, Miloš ; Zheng, Riliang ; Canabarro, Carla..
Molecular Genetics and Metabolism. 135 (2022) 2 - p. S96-S97 , 2022

Link: https://doi.org/10.1016/..

12

Safety, Pharmacokinetics, and Pharmacodynamics of Oral Veng..:

Peterschmitt, M. Judith ; Saiki, Hidemoto ; Hatano, Taku...
Journal of Parkinson's Disease. 12 (2022) 2 - p. 557-570 , 2022

Link: https://doi.org/10.3233/..

13

Pharmacokinetics and biomarker responses in patients with G..:

Peterschmitt, M. Judith ; Sharma, Jyoti ; Karetsky, Lina...
Molecular Genetics and Metabolism. 135 (2022) 2 - p. S96 , 2022

Link: https://doi.org/10.1016/..

14

A study to evaluate the effect of venglustat on neuropathic..:

Hopkin, Robert ; Kupferman, Joseph ; Deegan, Patrick B....
Molecular Genetics and Metabolism. 135 (2022) 2 - p. S56-S57 , 2022

Link: https://doi.org/10.1016/..

15

Venglustat combined with imiglucerase for neurological dise..:

Schiffmann, Raphael ; Cox, Timothy M ; Dedieu, Jean-François...
Brain. 146 (2022) 2 - p. 461-474 , 2022

Link: https://doi.org/10.1093/..