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1.
Effect of avalglucosidase alfa on disease-specific and general patient-reported outcomes in treatment-naïve adults with late-onset Pompe disease compared with alglucosidase alfa: Meaningful change analyses from the Phase 3 COMET trial.
Mol Genet Metab
; 141(2): 108121, 2024 Feb.
Artículo
en Inglés
| MEDLINE | ID: mdl-38184428
2.
Effect of eliglustat on the pharmacokinetics of digoxin, metoprolol, and oral contraceptives and absorption of eliglustat when coadministered with acid-reducing agents.
Mol Genet Metab
; 129(4): 278-285, 2020 04.
Artículo
en Inglés
| MEDLINE | ID: mdl-32029355
3.
Idarucizumab for Reversing Dabigatran-Induced Anticoagulation: A Systematic Review.
Am J Ther
; 25(3): e333-e338, 2018.
Artículo
en Inglés
| MEDLINE | ID: mdl-27175894
4.
Short stature as a presenting symptom of attenuated Mucopolysaccharidosis type I: case report and clinical insights.
BMC Endocr Disord
; 18(1): 83, 2018 Nov 12.
Artículo
en Inglés
| MEDLINE | ID: mdl-30419879
5.
Applying the win ratio method in clinical trials of orphan drugs: an analysis of data from the COMET trial of avalglucosidase alfa in patients with late-onset Pompe disease.
Orphanet J Rare Dis
; 19(1): 14, 2024 Jan 12.
Artículo
en Inglés
| MEDLINE | ID: mdl-38216959
6.
Post-hoc Nonparametric Analysis of Forced Vital Capacity in the COMET Trial Demonstrates Superiority of Avalglucosidase Alfa vs Alglucosidase Alfa.
J Neuromuscul Dis
; 11(2): 369-374, 2024.
Artículo
en Inglés
| MEDLINE | ID: mdl-38160363
7.
Efficacy and Safety of Avalglucosidase Alfa in Patients With Late-Onset Pompe Disease After 97 Weeks: A Phase 3 Randomized Clinical Trial.
JAMA Neurol
; 80(6): 558-567, 2023 06 01.
Artículo
en Inglés
| MEDLINE | ID: mdl-37036722
8.
Safety and efficacy of avalglucosidase alfa versus alglucosidase alfa in patients with late-onset Pompe disease (COMET): a phase 3, randomised, multicentre trial.
Lancet Neurol
; 20(12): 1012-1026, 2021 12.
Artículo
en Inglés
| MEDLINE | ID: mdl-34800399
9.
Effects of paroxetine, ketoconazole, and rifampin on the metabolism of eliglustat, an oral substrate reduction therapy for Gaucher disease type 1.
Mol Genet Metab Rep
; 22: 100552, 2020 Mar.
Artículo
en Inglés
| MEDLINE | ID: mdl-31993325
10.
Respiratory function during enzyme replacement therapy in late-onset Pompe disease: longitudinal course, prognostic factors, and the impact of time from diagnosis to treatment start.
J Neurol
; 267(10): 3038-3053, 2020 Oct.
Artículo
en Inglés
| MEDLINE | ID: mdl-32524257
11.
The Latin American experience with a next generation sequencing genetic panel for recessive limb-girdle muscular weakness and Pompe disease.
Orphanet J Rare Dis
; 15(1): 11, 2020 01 13.
Artículo
en Inglés
| MEDLINE | ID: mdl-31931849
12.
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