Effect of Oral Eliglustat on Splenomegaly in Patients With Gaucher Disease Type 1
نویسندگان
چکیده
منابع مشابه
Effect of oral eliglustat on splenomegaly in patients with Gaucher disease type 1: the ENGAGE randomized clinical trial.
IMPORTANCE Gaucher disease type 1 is characterized by hepatosplenomegaly, anemia, thrombocytopenia, and skeletal disease. A safe, effective oral therapy is needed. OBJECTIVE To determine whether eliglustat, a novel oral substrate reduction therapy, safely reverses clinical manifestations in untreated adults with Gaucher disease type 1. DESIGN, SETTING, AND PARTICIPANTS Phase 3, randomized, ...
متن کاملEliglustat tartrate for the treatment of adults with type 1 Gaucher disease
The purpose of this article is to review eliglustat tartrate, a substrate reduction therapy, for the treatment of Gaucher disease type 1 (GD1). GD is an rare inborn error of metabolism caused by accumulation of lipid substrates such as glucosylceramide within the monocyte-macrophage system that affects the body by causing enlargement of the spleen and liver, destruction of bone, and abnormaliti...
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In the phase 3 Study of Eliglustat Tartrate (Genz-112638) in Patients With Gaucher Disease Who Have Reached Therapeutic Goals With Enzyme Replacement Therapy (ENCORE), at 1 year, eliglustat was noninferior to imiglucerase enzyme therapy in maintaining stable platelet counts, hemoglobin concentrations, and spleen and liver volumes. After this primary analysis period, patients entered a long-term...
متن کاملEliglustat tartrate for the treatment of adults with type 1 Gaucher disease [Corrigendum]
[This corrects the article on p. 4639 in vol. 9.].
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ژورنال
عنوان ژورنال: JAMA
سال: 2015
ISSN: 0098-7484
DOI: 10.1001/jama.2015.459