Detalhe da pesquisa
1.
Dystrophin and mini-dystrophin quantification by mass spectrometry in skeletal muscle for gene therapy development in Duchenne muscular dystrophy.
Gene Ther
; 29(10-11): 608-615, 2022 11.
Artigo
em Inglês
| MEDLINE | ID: mdl-34737451
2.
Phase 1b Study to Evaluate Safety, Tolerability, and Maximum Tolerated Dose of PF-05230907 for Intracerebral Hemorrhage.
Stroke
; 52(1): 294-298, 2021 01.
Artigo
em Inglês
| MEDLINE | ID: mdl-33272131
3.
Towards regulatory endorsement of drug development tools to promote the application of model-informed drug development in Duchenne muscular dystrophy.
J Pharmacokinet Pharmacodyn
; 46(5): 441-455, 2019 10.
Artigo
em Inglês
| MEDLINE | ID: mdl-31127458
4.
Development and Validation of a Western Blot Method to Quantify Mini-Dystrophin in Human Skeletal Muscle Biopsies.
AAPS J
; 25(1): 12, 2022 12 20.
Artigo
em Inglês
| MEDLINE | ID: mdl-36539515
5.
Reducing Patient Burden in Clinical Trials Through the Use of Historical Controls: Appropriate Selection of Historical Data to Minimize Risk of Bias.
Ther Innov Regul Sci
; 54(4): 850-860, 2020 07.
Artigo
em Inglês
| MEDLINE | ID: mdl-32557308
6.
Minimizing Patient Burden Through the Use of Historical Subject-Level Data in Innovative Confirmatory Clinical Trials: Review of Methods and Opportunities.
Ther Innov Regul Sci
; 52(5): 546-559, 2018 09.
Artigo
em Inglês
| MEDLINE | ID: mdl-29909645
7.
Optimal stratification in outcome prediction using baseline information.
Biometrika
; 103(4): 817-828, 2016 12.
Artigo
em Inglês
| MEDLINE | ID: mdl-29422691
8.
Efavirenz liquid formulation in human immunodeficiency virus-infected children.
Pediatr Infect Dis J
; 21(7): 659-63, 2002 Jul.
Artigo
em Inglês
| MEDLINE | ID: mdl-12237599
9.
A retrospective, multinational, multicenter study on the natural history of infantile-onset Pompe disease.
J Pediatr
; 148(5): 671-676, 2006 May.
Artigo
em Inglês
| MEDLINE | ID: mdl-16737883
10.
T69D/N pol mutation, human immunodeficiency virus type 1 RNA levels, and syncytium-inducing phenotype are associated with CD4 cell depletion during didanosine therapy.
J Infect Dis
; 185(4): 448-55, 2002 Feb 15.
Artigo
em Inglês
| MEDLINE | ID: mdl-11865396
11.
Intrapatient variability of efavirenz concentrations as a predictor of virologic response to antiretroviral therapy.
Antimicrob Agents Chemother
; 48(3): 979-84, 2004 Mar.
Artigo
em Inglês
| MEDLINE | ID: mdl-14982792