32 results on '"Minini, Pascal"'
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2. Venglustat in GBA1-related Parkinson's disease – Authors' reply
3. Safety and efficacy of venglustat in GBA1-associated Parkinson's disease: an international, multicentre, double-blind, randomised, placebo-controlled, phase 2 trial
4. 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
5. Two years of venglustat combined with imiglucerase shows continued positive effects on neurological features and brain connectivity in adults with Gaucher disease type 3
6. 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
7. Venglustat, a novel brain-penetrant glucosylceramide synthase inhibitor, for Gaucher disease type 3: Phase 3 LEAP2MONO trial design
8. 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
9. AMETHIST: Baseline characteristics from a phase 3 trial of venglustat, a novel brain-penetrant glucosylceramide synthase inhibitor, in GM2-gangliosidoses and related diseases
10. 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
11. Venglustat, a novel brain-penetrant glucosylceramide synthase inhibitor, for GM2 gangliosidosis and related diseases: Phase 3 AMETHIST trial design
12. Pharmacokinetics and biomarker responses in patients with Gaucher disease type 3 or -associated Parkinson's disease treated with venglustat
13. A study to evaluate the effect of venglustat on neuropathic and abdominal pain in symptomatic adult patients with Fabry disease
14. Oral venglustat in Parkinson disease patients with a mutation: Study design of part 2 of the MOVES-PD trial and patient characteristics
15. Safety, Pharmacokinetics, and Pharmacodynamics of Oral Venglustat in Patients With Parkinson's Disease and a GBA Mutation: Results From Part 1 of the Randomised, Double-Blinded, Placebo-Controlled MOVES-PD Trial
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. Lipoprotein(a) reductions from PCSK9 inhibition and major adverse cardiovascular events: Pooled analysis of alirocumab phase 3 trials
19. Venglustat in adult Gaucher disease type 3: Preliminary safety, pharmacology, and exploratory efficacy from a phase 2 trial in combination with imiglucerase (LEAP)
20. Safety, tolerability and pharmacokinetics of oral venglustat in Parkinson disease patients with a GBA mutation
21. Efficacy and safety of the PCSK9 monoclonal antibody alirocumab versus placebo with heterozygous familial hypercholesterolemia
22. Functional performance in late-onset GM2 gangliosidosis (Tay-Sachs and Sandhoff diseases), longitudinal data over 3 consecutive years
23. Effect of Alirocumab on Lipoprotein(a) Over ≥1.5 Years (from the Phase 3 ODYSSEY Program)
24. Efficacy and safety of the proprotein convertase subtilisin/kexin type 9 monoclonal antibody alirocumab vs placebo in patients with heterozygous familial hypercholesterolemia
25. Efficacy of alirocumab in high cardiovascular risk populations with or without heterozygous familial hypercholesterolemia: Pooled analysis of eight ODYSSEY Phase 3 clinical program trials
26. Inverse Relationship Between Major Adverse Cardiovascular Events and non-High-Density Lipoprotein Cholesterol and Apolipoprotein B in Phase 3 Trials of Alirocumab
27. RELATIONSHIP BETWEEN PERCENTAGE REDUCTION IN LOW-DENSITY LIPOPROTEIN CHOLESTEROL LEVELS AND MAJOR ATHEROSCLEROTIC CARDIOVASCULAR DISEASE AMONG PATIENTS TREATED WITH STATINS +/- ALIROCUMAB OR EZETIMIBE IN THE PHASE 3 ODYSSEY TRIALS
28. RELATIONSHIP BETWEEN MAJOR ADVERSE CARDIOVASCULAR EVENTS AND ACHIEVED LOW-DENSITY LIPOPROTEIN CHOLESTEROL LEVELS IN PHASE 3 ODYSSEY TRIALS OF ALIROCUMAB VERSUS CONTROL
29. EFFICACY AND SAFETY OF ALIROCUMAB STRATIFIED BY AGE IN PHASE 3 TRIALS
30. Bleeding Risk in Patients With Atrial Fibrillation
31. Effects of a selective vasopressin V2 receptor antagonist, satavaptan, on ascites recurrence after paracentesis in patients with cirrhosis
32. NET CLINICAL BENEFIT OF COMBINATION ANTICOAGULANT AND ANTIPLATELET THERAPY VERSUS ANTICOAGULATION ALONE IN ATRIAL FIBRILLATION PATIENTS: RESULTS FROM THE AMADEUS TRIAL
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