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1.
Outcomes of screening for gammopathies in children and adults with Gaucher disease type 1 in a cohort from Brazil and the United States.
Am J Med Genet C Semin Med Genet
; 184(4): 1052-1059, 2020 12.
Artículo
en Inglés
| MEDLINE | ID: mdl-33277783
2.
Eliglustat maintains long-term clinical stability in patients with Gaucher disease type 1 stabilized on enzyme therapy.
Blood
; 129(17): 2375-2383, 2017 04 27.
Artículo
en Inglés
| MEDLINE | ID: mdl-28167660
3.
Transformation in pretreatment manifestations of Gaucher disease type 1 during two decades of alglucerase/imiglucerase enzyme replacement therapy in the International Collaborative Gaucher Group (ICGG) Gaucher Registry.
Am J Hematol
; 92(9): 929-939, 2017 Sep.
Artículo
en Inglés
| MEDLINE | ID: mdl-28569047
4.
Eliglustat compared with imiglucerase in patients with Gaucher's disease type 1 stabilised on enzyme replacement therapy: a phase 3, randomised, open-label, non-inferiority trial.
Lancet
; 385(9985): 2355-62, 2015 Jun 13.
Artículo
en Inglés
| MEDLINE | ID: mdl-25819691
5.
Recommendations for the use of eliglustat in the treatment of adults with Gaucher disease type 1 in the United States.
Mol Genet Metab
; 117(2): 95-103, 2016 Feb.
Artículo
en Inglés
| MEDLINE | ID: mdl-26387627
6.
Glycogen storage disease type IV: novel mutations and molecular characterization of a heterogeneous disorder.
J Inherit Metab Dis
; 33 Suppl 3: S83-90, 2010 Dec.
Artículo
en Inglés
| MEDLINE | ID: mdl-20058079
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