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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. 2018 Feb;123(2):69-75. doi: 10.1016/j.ymgme.2018.01.002. Epub 2018 Jan 5.
Mol Genet Metab. 2018.
PMID: 29336994
Review.
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.
Sampayo-Cordero M, Miguel-Huguet B, Pardo-Mateos A, Malfettone A, Pérez-García J, Llombart-Cussac A, Cortés J, Moltó-Abad M, Muñoz-Delgado C, Pérez-Quintana M, Pérez-López J.
Sampayo-Cordero M, et al. Among authors: miguel huguet b.
Orphanet J Rare Dis. 2019 Oct 21;14(1):230. doi: 10.1186/s13023-019-1202-6.
Orphanet J Rare Dis. 2019.
PMID: 31639024
Free PMC article.
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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).
Sampayo-Cordero M, Miguel-Huguet B, Malfettone A, Pérez-García JM, Llombart-Cussac A, Cortés J, Pardo A, Pérez-López J.
Sampayo-Cordero M, et al. Among authors: miguel huguet b.
Int J Environ Res Public Health. 2020 Sep 10;17(18):6590. doi: 10.3390/ijerph17186590.
Int J Environ Res Public Health. 2020.
PMID: 32927819
Free PMC article.
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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".
Sampayo-Cordero M, Miguel-Huguet B, Malfettone A, Pérez-García JM, Llombart-Cussac A, Cortés J, Pardo A, Pérez-López J.
Sampayo-Cordero M, et al. Among authors: miguel huguet b.
Front Mol Biosci. 2021 Jun 22;8:690615. doi: 10.3389/fmolb.2021.690615. eCollection 2021.
Front Mol Biosci. 2021.
PMID: 34239895
Free PMC article.
Review.
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Inclusion of non-inferiority analysis in superiority-based clinical trials with single-arm, two-stage Simon's design.
Sampayo-Cordero M, Miguel-Huguet B, Pérez-García J, Páez D, Guerrero-Zotano ÁL, Garde-Noguera J, Aguirre E, Holgado E, López-Miranda E, Huang X, Malfettone A, Llombart-Cussac A, Cortés J.
Sampayo-Cordero M, et al. Among authors: miguel huguet b.
Contemp Clin Trials Commun. 2020 Nov 28;20:100678. doi: 10.1016/j.conctc.2020.100678. eCollection 2020 Dec.
Contemp Clin Trials Commun. 2020.
PMID: 33336109
Free PMC article.
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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.
Sampayo-Cordero M, Miguel-Huguet B, Malfettone A, López-Miranda E, Gion M, Abad E, Alcalá-López D, Pérez-Escuredo J, Pérez-García JM, Llombart-Cussac A, Cortés J.
Sampayo-Cordero M, et al. Among authors: miguel huguet b.
Front Oncol. 2023 Jul 11;13:1048242. doi: 10.3389/fonc.2023.1048242. eCollection 2023.
Front Oncol. 2023.
PMID: 37496662
Free PMC article.
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Transanal Hemorrhoidal Dearterialization With Mucopexy Versus Vessel-Sealing Device Hemorrhoidectomy for Grade III to IV Hemorrhoids: Long-term Outcomes From the THDLIGA Randomized Controlled Trial.
Trenti L, Biondo S, Espin-Basany E, Barrios O, Sanchez-Garcia JL, Landaluce-Olavarria A, Bermejo-Marcos E, Garcia-Martinez MT, Alias Jimenez D, Jimenez F, Alonso A, Manso MB, Kreisler E; THDLIGA-RCT Study Group.
Trenti L, et al.
Dis Colon Rectum. 2023 Aug 1;66(8):e818-e825. doi: 10.1097/DCR.0000000000002272. Epub 2022 Mar 1.
Dis Colon Rectum. 2023.
PMID: 35239526
Clinical Trial.
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