Detalhe da pesquisa
1.
Mitochondrial modulation with leriglitazone as a potential treatment for Rett syndrome.
J Transl Med
; 21(1): 756, 2023 10 26.
Artigo
em Inglês
| MEDLINE | ID: mdl-37884937
2.
Development of PPARγ Agonists for the Treatment of Neuroinflammatory and Neurodegenerative Diseases: Leriglitazone as a Promising Candidate.
Int J Mol Sci
; 24(4)2023 Feb 06.
Artigo
em Inglês
| MEDLINE | ID: mdl-36834611
3.
PPAR gamma agonist leriglitazone improves frataxin-loss impairments in cellular and animal models of Friedreich Ataxia.
Neurobiol Dis
; 148: 105162, 2021 01.
Artigo
em Inglês
| MEDLINE | ID: mdl-33171227
4.
Clinical pharmacokinetics of leriglitazone and a translational approach using PBPK modeling to guide the selection of the starting dose in children.
CPT Pharmacometrics Syst Pharmacol
; 13(6): 982-993, 2024 Jun.
Artigo
em Inglês
| MEDLINE | ID: mdl-38549500
5.
PPAR Gamma Agonist Leriglitazone Recovers Alterations Due to Pank2-Deficiency in hiPS-Derived Astrocytes.
Pharmaceutics
; 15(1)2023 Jan 06.
Artigo
em Inglês
| MEDLINE | ID: mdl-36678831
6.
Safety and efficacy of leriglitazone for preventing disease progression in men with adrenomyeloneuropathy (ADVANCE): a randomised, double-blind, multi-centre, placebo-controlled phase 2-3 trial.
Lancet Neurol
; 22(2): 127-136, 2023 02.
Artigo
em Inglês
| MEDLINE | ID: mdl-36681445
7.
Characterisation of two deletions involving NPC1 and flanking genes in Niemann-Pick type C disease patients.
Mol Genet Metab
; 107(4): 716-20, 2012 Dec.
Artigo
em Inglês
| MEDLINE | ID: mdl-23142039
8.
The brain penetrant PPARγ agonist leriglitazone restores multiple altered pathways in models of X-linked adrenoleukodystrophy.
Sci Transl Med
; 13(596)2021 06 02.
Artigo
em Inglês
| MEDLINE | ID: mdl-34078742
9.
Present and future of antisense therapy for splicing modulation in inherited metabolic disease.
J Inherit Metab Dis
; 33(4): 397-403, 2010 Aug.
Artigo
em Inglês
| MEDLINE | ID: mdl-20577904
10.
Antisense oligonucleotide treatment for a pseudoexon-generating mutation in the NPC1 gene causing Niemann-Pick type C disease.
Hum Mutat
; 30(11): E993-E1001, 2009 Nov.
Artigo
em Inglês
| MEDLINE | ID: mdl-19718781
11.
Identification and characterization of SMPD1 mutations causing Niemann-Pick types A and B in Spanish patients.
Hum Mutat
; 30(7): 1117-22, 2009 Jul.
Artigo
em Inglês
| MEDLINE | ID: mdl-19405096
12.
Pharmacological chaperones for enzyme enhancement therapy in genetic diseases.
Pharm Pat Anal
; 2(1): 109-24, 2013 Jan.
Artigo
em Inglês
| MEDLINE | ID: mdl-24236974
13.
Generation of a human neuronal stable cell model for niemann-pick C disease by RNA interference.
JIMD Rep
; 4: 29-37, 2012.
Artigo
em Inglês
| MEDLINE | ID: mdl-23430894