Eliglustat maintains long-term clinical stability in patients with Gaucher disease type 1 stabilized on enzyme therapy.
Sign Up to like & getrecommendations! Published in 2017 at "Blood"
DOI: 10.1182/blood-2016-12-758409
Abstract: 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… read more here.
Keywords: patients gaucher; gaucher disease; term; long term ... See more keywords
New correlations between ocular parameters and disease severity in Spanish patients with Gaucher’s disease Type I
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DOI: 10.1371/journal.pone.0260241
Abstract: Background Gaucher’s disease is associated with a high variety of structural and functional abnormalities in the eye, which do not always affect visual acuity. The purpose of this study was to analyse ocular features in… read more here.
Keywords: patients gaucher; thickness; disease type; disease severity ... See more keywords