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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.
Artículo
en Inglés
| MEDLINE | ID: mdl-37386786
2.
Clinical studies in lysosomal storage diseases: past, present and future.
Pediatr Endocrinol Rev;
11 Suppl 1: 68-76, 2013 Nov.
Artículo
en Inglés
| MEDLINE | ID: mdl-24380125
3.
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.
Artículo
en Inglés
| MEDLINE | ID: mdl-28160230
4.
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.
Artículo
en Inglés
| MEDLINE | ID: mdl-27980008
5.
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.
Artículo
en Inglés
| MEDLINE | ID: mdl-28818518
6.
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.
Artículo
en Inglés
| MEDLINE | ID: mdl-16769255
7.
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.
Artículo
en Inglés
| MEDLINE | ID: mdl-26989892
8.
Gene therapy as a new treatment option for inherited monogenic diseases.
Eur J Intern Med;
25(1): 31-6, 2014 Jan.
Artículo
en Inglés
| MEDLINE | ID: mdl-24129166
9.
Clinical studies in lysosomal storage diseases: Past, present, and future.
Rare Dis;
1: e26690, 2013.
Artículo
en Inglés
| MEDLINE | ID: mdl-25003011
10.
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.
Artículo
en Inglés
| MEDLINE | ID: mdl-23176611
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