21 results on '"Minini, Pascal"'
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2. 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
3. The STAGED-PKD 2-Stage Adaptive Study With a Patient Enrichment Strategy and Treatment Effect Modeling for Improved Study Design Efficiency in Patients With ADPKD
4. Efficacy and safety of the proprotein convertase subtilisin/kexin type 9 monoclonal antibody alirocumab vs placebo in patients with heterozygous familial hypercholesterolemia
5. Effect of Alirocumab on Lipoprotein(a) Over ≥1.5 Years (from the Phase 3 ODYSSEY Program)
6. AMETHIST: Baseline characteristics from a phase 3 trial of venglustat, a novel brain-penetrant glucosylceramide synthase inhibitor, in GM2-gangliosidoses and related diseases
7. 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
8. Venglustat, a novel brain-penetrant glucosylceramide synthase inhibitor, for Gaucher disease type 3: Phase 3 LEAP2MONO trial design
9. 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
10. Two years of venglustat combined with imiglucerase shows continued positive effects on neurological features and brain connectivity in adults with Gaucher disease type 3
11. Bleeding Risk in Patients With Atrial Fibrillation: The AMADEUS Study
12. Pharmacokinetics and biomarker responses in patients with Gaucher disease type 3 or GBA-associated Parkinson's disease treated with venglustat
13. Venglustat, a novel brain-penetrant glucosylceramide synthase inhibitor, for GM2 gangliosidosis and related diseases: Phase 3 AMETHIST trial design
14. A study to evaluate the effect of venglustat on neuropathic and abdominal pain in symptomatic adult patients with Fabry disease
15. Oral venglustat in Parkinson disease patients with a GBA mutation: Study design of part 2 of the MOVES-PD trial and patient characteristics
16. Natural history of late-onset GM2 gangliosidosis: Four years of data collected at NTSAD annual conferences
17. Safety, pharmacokinetics, and pharmacodynamics of oral venglustat in Parkinson disease patients with a GBA mutation from Japan and the rest of the world: Results from part 1 of the MOVES-PD study
18. Functional performance in late-onset GM2 gangliosidosis (Tay-Sachs and Sandhoff diseases), longitudinal data over 3 consecutive years
19. Inverse Relationship Between Major Adverse Cardiovascular Events and non-High-Density Lipoprotein Cholesterol and Apolipoprotein B in Phase 3 Trials of Alirocumab
20. Venglustat in adult Gaucher disease type 3: Preliminary safety, pharmacology, and exploratory efficacy from a phase 2 trial in combination with imiglucerase (LEAP)
21. Safety, tolerability and pharmacokinetics of oral venglustat in Parkinson disease patients with a GBA mutation
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