Detalhe da pesquisa
1.
Risdiplam-Treated Infants with Type 1 Spinal Muscular Atrophy versus Historical Controls.
N Engl J Med
; 385(5): 427-435, 2021 07 29.
Artigo
em Inglês
| MEDLINE | ID: mdl-34320287
2.
Risdiplam in Type 1 Spinal Muscular Atrophy.
N Engl J Med
; 384(10): 915-923, 2021 03 11.
Artigo
em Inglês
| MEDLINE | ID: mdl-33626251
3.
Risdiplam in types 2 and 3 spinal muscular atrophy: A randomised, placebo-controlled, dose-finding trial followed by 24 months of treatment.
Eur J Neurol
; 30(7): 1945-1956, 2023 07.
Artigo
em Inglês
| MEDLINE | ID: mdl-35837793
4.
Addressing Today's Absorption, Distribution, Metabolism, and Excretion (ADME) Challenges in the Translation of In Vitro ADME Characteristics to Humans: A Case Study of the SMN2 mRNA Splicing Modifier Risdiplam.
Drug Metab Dispos
; 50(1): 65-75, 2022 01.
Artigo
em Inglês
| MEDLINE | ID: mdl-34620695
5.
Effect of mild or moderate hepatic impairment on the pharmacokinetics of risdiplam.
Br J Clin Pharmacol
; 88(8): 3749-3759, 2022 08.
Artigo
em Inglês
| MEDLINE | ID: mdl-35301746
6.
Ocrelizumab in relapsing and primary progressive multiple sclerosis: Pharmacokinetic and pharmacodynamic analyses of OPERA I, OPERA II and ORATORIO.
Br J Clin Pharmacol
; 87(6): 2511-2520, 2021 06.
Artigo
em Inglês
| MEDLINE | ID: mdl-33202059
7.
A phase 1 healthy male volunteer single escalating dose study of the pharmacokinetics and pharmacodynamics of risdiplam (RG7916, RO7034067), a SMN2 splicing modifier.
Br J Clin Pharmacol
; 85(1): 181-193, 2019 01.
Artigo
em Inglês
| MEDLINE | ID: mdl-30302786
8.
A UGT2B10 splicing polymorphism common in african populations may greatly increase drug exposure.
J Pharmacol Exp Ther
; 352(2): 358-67, 2015 Feb.
Artigo
em Inglês
| MEDLINE | ID: mdl-25503386
9.
Effect of gastric pH on erlotinib pharmacokinetics in healthy individuals: omeprazole and ranitidine.
Anticancer Drugs
; 26(5): 565-72, 2015 Jun.
Artigo
em Inglês
| MEDLINE | ID: mdl-25643050
10.
JEWELFISH: 24-month results from an open-label study in non-treatment-naïve patients with SMA receiving treatment with risdiplam.
J Neurol
; 2024 May 11.
Artigo
em Inglês
| MEDLINE | ID: mdl-38733387
11.
Estimating drug concentration-response relationships by applying causal inference methods for continuous point exposures and time-to-event outcomes.
Stat Methods Med Res
; 32(12): 2440-2454, 2023 12.
Artigo
em Inglês
| MEDLINE | ID: mdl-37964549
12.
Estimation of FMO3 Ontogeny by Mechanistic Population Pharmacokinetic Modelling of Risdiplam and Its Impact on Drug-Drug Interactions in Children.
Clin Pharmacokinet
; 62(6): 891-904, 2023 06.
Artigo
em Inglês
| MEDLINE | ID: mdl-37148485
13.
Association of Higher Ocrelizumab Exposure With Reduced Disability Progression in Multiple Sclerosis.
Neurol Neuroimmunol Neuroinflamm
; 10(2)2023 03.
Artigo
em Inglês
| MEDLINE | ID: mdl-36792367
14.
Risdiplam in Patients Previously Treated with Other Therapies for Spinal Muscular Atrophy: An Interim Analysis from the JEWELFISH Study.
Neurol Ther
; 12(2): 543-557, 2023 Apr.
Artigo
em Inglês
| MEDLINE | ID: mdl-36780114
15.
Two-year efficacy and safety of risdiplam in patients with type 2 or non-ambulant type 3 spinal muscular atrophy (SMA).
J Neurol
; 270(5): 2531-2546, 2023 May.
Artigo
em Inglês
| MEDLINE | ID: mdl-36735057
16.
Functional improvement in mouse models of familial amyotrophic lateral sclerosis by PEGylated insulin-like growth factor I treatment depends on disease severity.
Amyotroph Lateral Scler
; 13(5): 418-29, 2012 Sep.
Artigo
em Inglês
| MEDLINE | ID: mdl-22871074
17.
Safety and efficacy of risdiplam in patients with type 1 spinal muscular atrophy (FIREFISH part 2): secondary analyses from an open-label trial.
Lancet Neurol
; 21(12): 1110-1119, 2022 12.
Artigo
em Inglês
| MEDLINE | ID: mdl-36244364
18.
Safety and efficacy of once-daily risdiplam in type 2 and non-ambulant type 3 spinal muscular atrophy (SUNFISH part 2): a phase 3, double-blind, randomised, placebo-controlled trial.
Lancet Neurol
; 21(1): 42-52, 2022 01.
Artigo
em Inglês
| MEDLINE | ID: mdl-34942136
19.
Bioavailability and pharmacokinetic profile of balovaptan, a selective, brain-penetrant vasopressin 1a receptor antagonist, in healthy volunteers.
Expert Opin Investig Drugs
; 30(8): 893-901, 2021 Aug.
Artigo
em Inglês
| MEDLINE | ID: mdl-34176392
20.
Model-Based Drug-Drug Interaction Extrapolation Strategy From Adults to Children: Risdiplam in Pediatric Patients With Spinal Muscular Atrophy.
Clin Pharmacol Ther
; 110(6): 1547-1557, 2021 12.
Artigo
em Inglês
| MEDLINE | ID: mdl-34347881