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Genetics in Medicine : Official Journal of the American College of Medical Genetics|August 27, 2023
Long-term safety and efficacy of pegunigalsidase alfa: A multicenter 6-year study in adult patients with Fabry diseaseDerralynn Hughes, Derlis Gonzalez, Gustavo Maegawa, et al.Parkinsonism & Related Disorders|August 28, 2021
Pediatric Gaucher disease with intermediate type 2-3 phenotype associated with parkinsonian features and levodopa responsivenessAlejandra Darling, Pilar Irún, Pilar Giraldo, et al.Blood Cells, Molecules & Diseases|June 22, 2014
A Phase 3, multicenter, open-label, switchover trial to assess the safety and efficacy of taliglucerase alfa, a plant cell-expressed recombinant human glucocerebrosidase, in adult and pediatric patients with Gaucher disease previously treated with imigluceraseGregory M Pastores, Milan Petakov, Pilar Giraldo, et al.Cancer Research|April 17, 2004
NIN, a gene encoding a CEP110-like centrosomal protein, is fused to PDGFRB in a patient with a t(5;14)(q33;q24) and an imatinib-responsive myeloproliferative disorderJosé L Vizmanos, Francisco J Novo, José P Román, et al.Leukemia|November 25, 2023
Impact of age and comorbidities on the efficacy and tolerability of bosutinib in previously treated patients with chronic myeloid leukemia: results from the phase 4 BYOND studyGianantonio Rosti, Tim H Brümmendorf, Björn T Gjertsen, et al.Journal of Clinical Medicine|April 14, 2026
Improvement of Bone Mineral Density in Patients with Type 1 Gaucher Disease Treated with Velaglucerase Alfa: Results from Clinical StudiesAri Zimran, Jaco Botha, Richard Eastell, et al.Cytometry. Part B, Clinical Cytometry|December 7, 2007
Impact of trisomy 12, del(13q), del(17p), and del(11q) on the immunophenotype, DNA ploidy status, and proliferative rate of leukemic B-cells in chronic lymphocytic leukemiaSandra Quijano, Antonio López, Ana Rasillo, et al.Molecular Genetics and Metabolism Reports|October 11, 2016
Stability is maintained in adults with Gaucher disease type 1 switched from velaglucerase alfa to eliglustat or imiglucerase: A sub-analysis of the eliglustat ENCORE trialRebecca Pleat, Timothy M Cox, T Andrew Burrow, et al.Journal of Clinical Laboratory Analysis|August 6, 2024
Frequency of rs1051338 and rs116928232 Variants in Individuals from Northwest MexicoAngélica Alejandra Hernández-Orozco, Lennon Melendez-Aranda, Sandra Del Carmen Mendoza-Ruvalcaba, et al.Orphanet Journal of Rare Diseases|October 21, 2023
Safety and efficacy of pegunigalsidase alfa in patients with Fabry disease who were previously treated with agalsidase alfa: results from BRIDGE, a phase 3 open-label studyAleš Linhart, Gabriela Dostálová, Kathy Nicholls, et al.Pageof 16