Detalhe da pesquisa
1.
Evaluating longitudinal therapy effects via the North Star Ambulatory Assessment.
Muscle Nerve
; 64(5): 614-619, 2021 11.
Artigo
em Inglês
| MEDLINE | ID: mdl-34383312
2.
Deflazacort vs prednisone treatment for Duchenne muscular dystrophy: A meta-analysis of disease progression rates in recent multicenter clinical trials.
Muscle Nerve
; 61(1): 26-35, 2020 01.
Artigo
em Inglês
| MEDLINE | ID: mdl-31599456
3.
Ataluren in patients with nonsense mutation Duchenne muscular dystrophy (ACT DMD): a multicentre, randomised, double-blind, placebo-controlled, phase 3 trial.
Lancet
; 390(10101): 1489-1498, 2017 09 23.
Artigo
em Inglês
| MEDLINE | ID: mdl-28728956
4.
Deflazacort versus prednisone/prednisolone for maintaining motor function and delaying loss of ambulation: A post HOC analysis from the ACT DMD trial.
Muscle Nerve
; 58(5): 639-645, 2018 11.
Artigo
em Inglês
| MEDLINE | ID: mdl-30028519
5.
Ataluren treatment of patients with nonsense mutation dystrophinopathy.
Muscle Nerve
; 50(4): 477-87, 2014 Oct.
Artigo
em Inglês
| MEDLINE | ID: mdl-25042182
6.
Corticosteroids in Duchenne muscular dystrophy: major variations in practice.
Muscle Nerve
; 48(1): 27-31, 2013 Jul.
Artigo
em Inglês
| MEDLINE | ID: mdl-23483575
7.
The 6-minute walk test and other clinical endpoints in duchenne muscular dystrophy: reliability, concurrent validity, and minimal clinically important differences from a multicenter study.
Muscle Nerve
; 48(3): 357-68, 2013 Sep.
Artigo
em Inglês
| MEDLINE | ID: mdl-23674289
8.
The 6-minute walk test and other endpoints in Duchenne muscular dystrophy: longitudinal natural history observations over 48 weeks from a multicenter study.
Muscle Nerve
; 48(3): 343-56, 2013 Sep.
Artigo
em Inglês
| MEDLINE | ID: mdl-23681930
9.
Ataluren (PTC124) induces cystic fibrosis transmembrane conductance regulator protein expression and activity in children with nonsense mutation cystic fibrosis.
Am J Respir Crit Care Med
; 182(10): 1262-72, 2010 Nov 15.
Artigo
em Inglês
| MEDLINE | ID: mdl-20622033
10.
Meta-analyses of deflazacort versus prednisone/prednisolone in patients with nonsense mutation Duchenne muscular dystrophy.
J Comp Eff Res
; 10(18): 1337-1347, 2021 12.
Artigo
em Inglês
| MEDLINE | ID: mdl-34693725
11.
The 6-minute walk test in Duchenne/Becker muscular dystrophy: longitudinal observations.
Muscle Nerve
; 42(6): 966-74, 2010 Dec.
Artigo
em Inglês
| MEDLINE | ID: mdl-21038378
12.
The 6-minute walk test as a new outcome measure in Duchenne muscular dystrophy.
Muscle Nerve
; 41(4): 500-10, 2010 Apr.
Artigo
em Inglês
| MEDLINE | ID: mdl-19941337
13.
Longitudinal Evaluation of Working Memory in Duchenne Muscular Dystrophy.
J Clin Med
; 9(9)2020 Sep 11.
Artigo
em Inglês
| MEDLINE | ID: mdl-32933029
14.
Meta-analyses of ataluren randomized controlled trials in nonsense mutation Duchenne muscular dystrophy.
J Comp Eff Res
; 9(14): 973-984, 2020 10.
Artigo
em Inglês
| MEDLINE | ID: mdl-32851872
15.
Effectiveness of PTC124 treatment of cystic fibrosis caused by nonsense mutations: a prospective phase II trial.
Lancet
; 372(9640): 719-27, 2008 Aug 30.
Artigo
em Inglês
| MEDLINE | ID: mdl-18722008
16.
Ataluren use in patients with nonsense mutation Duchenne muscular dystrophy: patient demographics and characteristics from the STRIDE Registry.
J Comp Eff Res
; 8(14): 1187-1200, 2019 10.
Artigo
em Inglês
| MEDLINE | ID: mdl-31414621
17.
The relationship between deficit in digit span and genotype in nonsense mutation Duchenne muscular dystrophy.
Neurology
; 91(13): e1215-e1219, 2018 09 25.
Artigo
em Inglês
| MEDLINE | ID: mdl-30135256
18.
The minor gentamicin complex component, X2, is a potent premature stop codon readthrough molecule with therapeutic potential.
PLoS One
; 13(10): e0206158, 2018.
Artigo
em Inglês
| MEDLINE | ID: mdl-30359426
19.
Safety, tolerability, and pharmacokinetics of PTC124, a nonaminoglycoside nonsense mutation suppressor, following single- and multiple-dose administration to healthy male and female adult volunteers.
J Clin Pharmacol
; 47(4): 430-44, 2007 Apr.
Artigo
em Inglês
| MEDLINE | ID: mdl-17389552
20.
Phase 1 and pharmacokinetic study of intravenous irinotecan in refractory solid tumor patients with hepatic dysfunction.
Clin Cancer Res
; 12(12): 3782-91, 2006 Jun 15.
Artigo
em Inglês
| MEDLINE | ID: mdl-16778106