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 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
3.
PTC124 targets genetic disorders caused by nonsense mutations.
Nature
; 447(7140): 87-91, 2007 May 03.
Artigo
em Inglês
| MEDLINE | ID: mdl-17450125
4.
Disease progression rates in ambulatory Duchenne muscular dystrophy by steroid type, patient age and functional status.
J Comp Eff Res
; 12(4): e220190, 2023 04.
Artigo
em Inglês
| MEDLINE | ID: mdl-36749302
5.
Functional and Clinical Outcomes Associated with Steroid Treatment among Non-ambulatory Patients with Duchenne Muscular Dystrophy1.
J Neuromuscul Dis
; 10(1): 67-79, 2023.
Artigo
em Inglês
| MEDLINE | ID: mdl-36565131
6.
Safety and effectiveness of ataluren in patients with nonsense mutation DMD in the STRIDE Registry compared with the CINRG Duchenne Natural History Study (2015-2022): 2022 interim analysis.
J Neurol
; 270(8): 3896-3913, 2023 Aug.
Artigo
em Inglês
| MEDLINE | ID: mdl-37115359
7.
Natural History and Real-World Data in Rare Diseases: Applications, Limitations, and Future Perspectives.
J Clin Pharmacol
; 62 Suppl 2: S38-S55, 2022 12.
Artigo
em Inglês
| MEDLINE | ID: mdl-36461748
8.
Ataluren delays loss of ambulation and respiratory decline in nonsense mutation Duchenne muscular dystrophy patients.
J Comp Eff Res
; 11(3): 139-155, 2022 02.
Artigo
em Inglês
| MEDLINE | ID: mdl-34791888
9.
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
10.
Longitudinal Evaluation of Working Memory in Duchenne Muscular Dystrophy.
J Clin Med
; 9(9)2020 Sep 11.
Artigo
em Inglês
| MEDLINE | ID: mdl-32933029
11.
Safety and effectiveness of ataluren: comparison of results from the STRIDE Registry and CINRG DMD Natural History Study.
J Comp Eff Res
; 9(5): 341-360, 2020 04.
Artigo
em Inglês
| MEDLINE | ID: mdl-31997646
12.
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
13.
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
14.
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
15.
Correction to: Safety and effectiveness of ataluren in patients with nonsense mutation DMD in the STRIDE Registry compared with the CINRG Duchenne Natural History Study (2015-2022): 2022 interim analysis.
J Neurol
; 270(9): 4583, 2023 Sep.
Artigo
em Inglês
| MEDLINE | ID: mdl-37460854
16.
Discovery of Novel Small Molecule Inhibitors of VEGF Expression in Tumor Cells Using a Cell-Based High Throughput Screening Platform.
PLoS One
; 11(12): e0168366, 2016.
Artigo
em Inglês
| MEDLINE | ID: mdl-27992500
17.
Motor neuron disease. SMN2 splicing modifiers improve motor function and longevity in mice with spinal muscular atrophy.
Science
; 345(6197): 688-93, 2014 Aug 08.
Artigo
em Inglês
| MEDLINE | ID: mdl-25104390
18.
Identification of target messenger RNA substrates for the murine deleted in azoospermia-like RNA-binding protein.
Biol Reprod
; 66(2): 475-85, 2002 Feb.
Artigo
em Inglês
| MEDLINE | ID: mdl-11804965