Detalles de la búsqueda
1.
Head-to-head trial of pegunigalsidase alfa versus agalsidase beta in patients with Fabry disease and deteriorating renal function: results from the 2-year randomised phase III BALANCE study.
J Med Genet
; 2023 Nov 08.
Artículo
en Inglés
| MEDLINE | ID: mdl-37940383
2.
Long-term safety and efficacy of pegunigalsidase alfa: A multicenter 6-year study in adult patients with Fabry disease.
Genet Med
; 25(12): 100968, 2023 Dec.
Artículo
en Inglés
| MEDLINE | ID: mdl-37634127
3.
Serum Phospholipid Profile Changes in Gaucher Disease and Parkinson's Disease.
Int J Mol Sci
; 23(18)2022 Sep 08.
Artículo
en Inglés
| MEDLINE | ID: mdl-36142296
4.
Comparing the safety and efficacy of ruxolitinib in patients with Dynamic International Prognostic Scoring System low-, intermediate-1-, intermediate-2-, and high-risk myelofibrosis in JUMP, a Phase 3b, expanded-access study.
Hematol Oncol
; 39(4): 558-566, 2021 Oct.
Artículo
en Inglés
| MEDLINE | ID: mdl-34224180
5.
Maintenance therapy with ex vivo expanded lymphokine-activated killer cells and rituximab in patients with follicular lymphoma is safe and may delay disease progression.
Br J Haematol
; 189(6): 1064-1073, 2020 06.
Artículo
en Inglés
| MEDLINE | ID: mdl-32130737
6.
Primary analysis of JUMP, a phase 3b, expanded-access study evaluating the safety and efficacy of ruxolitinib in patients with myelofibrosis, including those with low platelet counts.
Br J Haematol
; 189(5): 888-903, 2020 06.
Artículo
en Inglés
| MEDLINE | ID: mdl-32017044
7.
Direct and indirect effects of the SARS-CoV-2 pandemic on Gaucher Disease patients in Spain: Time to reconsider home-based therapies?
Blood Cells Mol Dis
; 85: 102478, 2020 11.
Artículo
en Inglés
| MEDLINE | ID: mdl-32688219
8.
The impact of COVID-19 pandemic on the diagnosis and management of inborn errors of metabolism: A global perspective.
Mol Genet Metab
; 131(3): 285-288, 2020 11.
Artículo
en Inglés
| MEDLINE | ID: mdl-33004274
9.
LC-MS/MS analysis of plasma glucosylsphingosine as a biomarker for diagnosis and follow-up monitoring in Gaucher disease in the Spanish population.
Clin Chem Lab Med
; 58(5): 798-809, 2020 04 28.
Artículo
en Inglés
| MEDLINE | ID: mdl-32126008
10.
New variants in Spanish Niemann-Pick type c disease patients.
Mol Biol Rep
; 47(3): 2085-2095, 2020 Mar.
Artículo
en Inglés
| MEDLINE | ID: mdl-32060698
11.
Treatment of Fabry's Disease with the Pharmacologic Chaperone Migalastat.
N Engl J Med
; 375(6): 545-55, 2016 Aug 11.
Artículo
en Inglés
| MEDLINE | ID: mdl-27509102
12.
Long-term efficacy and safety results of taliglucerase alfa through 5years in adult treatment-naïve patients with Gaucher disease.
Blood Cells Mol Dis
; 78: 14-21, 2019 09.
Artículo
en Inglés
| MEDLINE | ID: mdl-27499018
13.
Clinical outcomes of patients with myelofibrosis after immediate transition to momelotinib from ruxolitinib.
Haematologica
; 109(2): 676-681, 2024 Feb 01.
Artículo
en Inglés
| MEDLINE | ID: mdl-37259556
14.
Safety and efficacy of bosutinib in fourth-line therapy of chronic myeloid leukemia patients.
Ann Hematol
; 98(2): 321-330, 2019 Feb.
Artículo
en Inglés
| MEDLINE | ID: mdl-30446802
15.
Pegunigalsidase alfa, a novel PEGylated enzyme replacement therapy for Fabry disease, provides sustained plasma concentrations and favorable pharmacodynamics: A 1-year Phase 1/2 clinical trial.
J Inherit Metab Dis
; 42(3): 534-544, 2019 05.
Artículo
en Inglés
| MEDLINE | ID: mdl-30834538
16.
The utility of magnetic resonance imaging for bone involvement in Gaucher disease. Assessing more than bone crises.
Blood Cells Mol Dis
; 68: 126-134, 2018 02.
Artículo
en Inglés
| MEDLINE | ID: mdl-27836527
17.
Long-term safety and efficacy of taliglucerase alfa in pediatric Gaucher disease patients who were treatment-naïve or previously treated with imiglucerase.
Blood Cells Mol Dis
; 68: 163-172, 2018 02.
Artículo
en Inglés
| MEDLINE | ID: mdl-27839981
18.
Twelve years of experience with miglustat in the treatment of type 1 Gaucher disease: The Spanish ZAGAL project.
Blood Cells Mol Dis
; 68: 173-179, 2018 02.
Artículo
en Inglés
| MEDLINE | ID: mdl-27836529
19.
Immune status of high-risk smoldering multiple myeloma patients and its therapeutic modulation under LenDex: a longitudinal analysis.
Blood
; 127(9): 1151-62, 2016 Mar 03.
Artículo
en Inglés
| MEDLINE | ID: mdl-26668134
20.
Demographics and patient characteristics of 1209 patients with Gaucher disease: Descriptive analysis from the Gaucher Outcome Survey (GOS).
Am J Hematol
; 93(2): 205-212, 2018 02.
Artículo
en Inglés
| MEDLINE | ID: mdl-29090476