Detalhe da pesquisa
1.
Agreement between the results of meta-analyses from case reports and from clinical studies regarding the efficacy of laronidase therapy in patients with mucopolysaccharidosis type I who initiated enzyme replacement therapy in adult age: An example of case reports meta-analyses as an useful tool for evidence-based medicine in rare diseases.
Mol Genet Metab
; 123(2): 69-75, 2018 02.
Artigo
em Inglês
| MEDLINE | ID: mdl-29336994
2.
A single-arm study design with non-inferiority and superiority time-to-event endpoints: a tool for proof-of-concept and de-intensification strategies in breast cancer.
Front Oncol
; 13: 1048242, 2023.
Artigo
em Inglês
| MEDLINE | ID: mdl-37496662
3.
The Impact of Excluding Nonrandomized Studies From Systematic Reviews in Rare Diseases: "The Example of Meta-Analyses Evaluating the Efficacy and Safety of Enzyme Replacement Therapy in Patients With Mucopolysaccharidosis".
Front Mol Biosci
; 8: 690615, 2021.
Artigo
em Inglês
| MEDLINE | ID: mdl-34239895
4.
The Value of Case Reports in Systematic Reviews from Rare Diseases. The Example of Enzyme Replacement Therapy (ERT) in Patients with Mucopolysaccharidosis Type II (MPS-II).
Int J Environ Res Public Health
; 17(18)2020 09 10.
Artigo
em Inglês
| MEDLINE | ID: mdl-32927819
5.
Inclusion of non-inferiority analysis in superiority-based clinical trials with single-arm, two-stage Simon's design.
Contemp Clin Trials Commun
; 20: 100678, 2020 Dec.
Artigo
em Inglês
| MEDLINE | ID: mdl-33336109
6.
Agreement between results of meta-analyses from case reports and clinical studies, regarding efficacy and safety of idursulfase therapy in patients with mucopolysaccharidosis type II (MPS-II). A new tool for evidence-based medicine in rare diseases.
Orphanet J Rare Dis
; 14(1): 230, 2019 10 21.
Artigo
em Inglês
| MEDLINE | ID: mdl-31639024
7.
The Impact of Excluding Nonrandomized Studies From Systematic Reviews in Rare Diseases: "The Example of Meta-Analyses Evaluating the Efficacy and Safety of Enzyme Replacement Therapy in Patients With Mucopolysaccharidosis"
Front. mol. biosci.
; : 690615, jun. 2021. graf, tab
Artigo
em Inglês
| Redbvs | ID: biblio-1418391