Detalhe da pesquisa
1.
Seladelpar efficacy and safety at 3 months in patients with primary biliary cholangitis: ENHANCE, a phase 3, randomized, placebo-controlled study.
Hepatology
; 78(2): 397-415, 2023 08 01.
Artigo
em Inglês
| MEDLINE | ID: mdl-37386786
2.
A phase II, randomized, open-label, 52-week study of seladelpar in patients with primary biliary cholangitis.
J Hepatol
; 77(2): 353-364, 2022 08.
Artigo
em Inglês
| MEDLINE | ID: mdl-35367282
3.
Duvoglustat HCl Increases Systemic and Tissue Exposure of Active Acid α-Glucosidase in Pompe Patients Co-administered with Alglucosidase α.
Mol Ther
; 25(5): 1199-1208, 2017 05 03.
Artigo
em Inglês
| MEDLINE | ID: mdl-28341561
4.
Oral pharmacological chaperone migalastat compared with enzyme replacement therapy in Fabry disease: 18-month results from the randomised phase III ATTRACT study.
J Med Genet
; 54(4): 288-296, 2017 04.
Artigo
em Inglês
| MEDLINE | ID: mdl-27834756
5.
Clinical studies in lysosomal storage diseases: past, present and future.
Pediatr Endocrinol Rev
; 11 Suppl 1: 68-76, 2013 Nov.
Artigo
em Inglês
| MEDLINE | ID: mdl-24380125
6.
Machine learning liver histology scores correlate with portal hypertension assessments in nonalcoholic steatohepatitis cirrhosis.
Aliment Pharmacol Ther
; 57(4): 409-417, 2023 02.
Artigo
em Inglês
| MEDLINE | ID: mdl-36647687
7.
A pharmacogenetic approach to identify mutant forms of α-galactosidase A that respond to a pharmacological chaperone for Fabry disease.
Hum Mutat
; 32(8): 965-77, 2011 Aug.
Artigo
em Inglês
| MEDLINE | ID: mdl-21598360
8.
Risk Evaluation and Mitigation Strategies (REMSs): Are They Improving Drug Safety? A Critical Review of REMSs Requiring Elements to Assure Safe Use (ETASU).
Drugs R D
; 17(2): 245-254, 2017 Jun.
Artigo
em Inglês
| MEDLINE | ID: mdl-28160230
9.
The Pharmacodynamics, Pharmacokinetics, and Safety of Arhalofenate in Combination with Febuxostat When Treating Hyperuricemia Associated with Gout.
J Rheumatol
; 44(3): 374-379, 2017 03.
Artigo
em Inglês
| MEDLINE | ID: mdl-27980008
10.
Seladelpar (MBX-8025), a selective PPAR-δ agonist, in patients with primary biliary cholangitis with an inadequate response to ursodeoxycholic acid: a double-blind, randomised, placebo-controlled, phase 2, proof-of-concept study.
Lancet Gastroenterol Hepatol
; 2(10): 716-726, 2017 10.
Artigo
em Inglês
| MEDLINE | ID: mdl-28818518
11.
The challenges of new drugs benefits and risks analysis: lessons from the ximelagatran FDA Cardiovascular Advisory Committee.
Contemp Clin Trials
; 27(5): 432-40, 2006 Oct.
Artigo
em Inglês
| MEDLINE | ID: mdl-16769255
12.
A Randomized, Double-Blind, Active- and Placebo-Controlled Efficacy and Safety Study of Arhalofenate for Reducing Flare in Patients With Gout.
Arthritis Rheumatol
; 68(8): 2027-34, 2016 08.
Artigo
em Inglês
| MEDLINE | ID: mdl-26989892
13.
An open-label study to determine the pharmacokinetics and safety of migalastat HCl in subjects with impaired renal function and healthy subjects with normal renal function.
Clin Pharmacol Drug Dev
; 4(4): 256-61, 2015 07.
Artigo
em Inglês
| MEDLINE | ID: mdl-27136905
14.
Oral Migalastat HCl Leads to Greater Systemic Exposure and Tissue Levels of Active α-Galactosidase A in Fabry Patients when Co-Administered with Infused Agalsidase.
PLoS One
; 10(8): e0134341, 2015.
Artigo
em Inglês
| MEDLINE | ID: mdl-26252393
15.
Gene therapy as a new treatment option for inherited monogenic diseases.
Eur J Intern Med
; 25(1): 31-6, 2014 Jan.
Artigo
em Inglês
| MEDLINE | ID: mdl-24129166
16.
Clinical studies in lysosomal storage diseases: Past, present, and future.
Rare Dis
; 1: e26690, 2013.
Artigo
em Inglês
| MEDLINE | ID: mdl-25003011
17.
Pharmacokinetics and Safety of Migalastat HCl and Effects on Agalsidase Activity in Healthy Volunteers.
Clin Pharmacol Drug Dev
; 2(2): 120-32, 2013 Apr.
Artigo
em Inglês
| MEDLINE | ID: mdl-27121667
18.
Migalastat HCl reduces globotriaosylsphingosine (lyso-Gb3) in Fabry transgenic mice and in the plasma of Fabry patients.
PLoS One
; 8(3): e57631, 2013.
Artigo
em Inglês
| MEDLINE | ID: mdl-23472096
19.
Novel quantitative method to evaluate globotriaosylceramide inclusions in renal peritubular capillaries by virtual microscopy in patients with fabry disease.
Arch Pathol Lab Med
; 136(7): 816-24, 2012 Jul.
Artigo
em Inglês
| MEDLINE | ID: mdl-22742555
20.
Safety and pharmacodynamic effects of a pharmacological chaperone on α-galactosidase A activity and globotriaosylceramide clearance in Fabry disease: report from two phase 2 clinical studies.
Orphanet J Rare Dis
; 7: 91, 2012 Nov 24.
Artigo
em Inglês
| MEDLINE | ID: mdl-23176611