Detalhe da pesquisa
1.
Durability of Voretigene Neparvovec for Biallelic RPE65-Mediated Inherited Retinal Disease: Phase 3 Results at 3 and 4 Years.
Ophthalmology
; 128(10): 1460-1468, 2021 10.
Artigo
em Inglês
| MEDLINE | ID: mdl-33798654
2.
Efficacy, Safety, and Durability of Voretigene Neparvovec-rzyl in RPE65 Mutation-Associated Inherited Retinal Dystrophy: Results of Phase 1 and 3 Trials.
Ophthalmology
; 126(9): 1273-1285, 2019 09.
Artigo
em Inglês
| MEDLINE | ID: mdl-31443789
3.
Efficacy and safety of voretigene neparvovec (AAV2-hRPE65v2) in patients with RPE65-mediated inherited retinal dystrophy: a randomised, controlled, open-label, phase 3 trial.
Lancet
; 390(10097): 849-860, 2017 Aug 26.
Artigo
em Inglês
| MEDLINE | ID: mdl-28712537
4.
Frequent source plasma donors are not at risk of iron depletion: the Ferritin Levels in Plasma Donor (FLIPD) study.
Transfusion
; 58(4): 951-959, 2018 04.
Artigo
em Inglês
| MEDLINE | ID: mdl-29520799
5.
A multicenter, retrospective medical record review of X-linked myotubular myopathy: The recensus study.
Muscle Nerve
; 57(4): 550-560, 2018 04.
Artigo
em Inglês
| MEDLINE | ID: mdl-29149770
6.
Novel mobility test to assess functional vision in patients with inherited retinal dystrophies.
Clin Exp Ophthalmol
; 46(3): 247-259, 2018 04.
Artigo
em Inglês
| MEDLINE | ID: mdl-28697537
7.
Cognitive endpoints for therapy development for neuronopathic mucopolysaccharidoses: Results of a consensus procedure.
Mol Genet Metab
; 121(2): 70-79, 2017 06.
Artigo
em Inglês
| MEDLINE | ID: mdl-28501294
8.
Enzyme replacement therapy for mucopolysaccharidosis VI: evaluation of long-term pulmonary function in patients treated with recombinant human N-acetylgalactosamine 4-sulfatase.
J Inherit Metab Dis
; 33(1): 51-60, 2010 Feb.
Artigo
em Inglês
| MEDLINE | ID: mdl-20140523
9.
Long-term follow-up of endurance and safety outcomes during enzyme replacement therapy for mucopolysaccharidosis VI: Final results of three clinical studies of recombinant human N-acetylgalactosamine 4-sulfatase.
Mol Genet Metab
; 94(4): 469-475, 2008 Aug.
Artigo
em Inglês
| MEDLINE | ID: mdl-18502162
10.
A simulation study of quantitative risk assessment for bivariate continuous outcomes.
Risk Anal
; 28(5): 1415-30, 2008 Oct.
Artigo
em Inglês
| MEDLINE | ID: mdl-18631306
11.
The impact of kidney function on outcomes following high risk myocardial infarction: findings from 27 610 patients.
Eur J Heart Fail
; 16(3): 289-99, 2014 Mar.
Artigo
em Inglês
| MEDLINE | ID: mdl-24464979
12.
Enzyme replacement therapy for mucopolysaccharidosis VI: Growth and pubertal development in patients treated with recombinant human N-acetylgalactosamine 4-sulfatase.
J Pediatr Rehabil Med
; 3(2): 89-100, 2010.
Artigo
em Inglês
| MEDLINE | ID: mdl-20634905
13.
Endovascular stent grafting versus open surgical repair of descending thoracic aortic aneurysms in low-risk patients: a multicenter comparative trial.
J Thorac Cardiovasc Surg
; 133(2): 369-77, 2007 Feb.
Artigo
em Inglês
| MEDLINE | ID: mdl-17258566
14.
Enzyme replacement therapy for mucopolysaccharidosis VI: a phase 3, randomized, double-blind, placebo-controlled, multinational study of recombinant human N-acetylgalactosamine 4-sulfatase (recombinant human arylsulfatase B or rhASB) and follow-on, open-label extension study.
J Pediatr
; 148(4): 533-539, 2006 Apr.
Artigo
em Inglês
| MEDLINE | ID: mdl-16647419
15.
Quantitative risk assessment for multivariate continuous outcomes with application to neurotoxicology: the bivariate case.
Biometrics
; 61(3): 757-66, 2005 Sep.
Artigo
em Inglês
| MEDLINE | ID: mdl-16135027
16.
Direct comparison of measures of endurance, mobility, and joint function during enzyme-replacement therapy of mucopolysaccharidosis VI (Maroteaux-Lamy syndrome): results after 48 weeks in a phase 2 open-label clinical study of recombinant human N-acetylgalactosamine 4-sulfatase.
Pediatrics
; 115(6): e681-9, 2005 Jun.
Artigo
em Inglês
| MEDLINE | ID: mdl-15930196