کد مقاله کد نشریه سال انتشار مقاله انگلیسی نسخه تمام متن
6225013 1607488 2011 8 صفحه PDF دانلود رایگان
عنوان انگلیسی مقاله ISI
Evaluation of Miglustat Treatment in Patients with Type III Mucopolysaccharidosis: A Randomized, Double-Blind, Placebo-Controlled Study
موضوعات مرتبط
علوم پزشکی و سلامت پزشکی و دندانپزشکی پریناتولوژی (پزشکی مادر و جنین)، طب اطفال و بهداشت کودک
پیش نمایش صفحه اول مقاله
Evaluation of Miglustat Treatment in Patients with Type III Mucopolysaccharidosis: A Randomized, Double-Blind, Placebo-Controlled Study
چکیده انگلیسی

ObjectiveTo evaluate the efficacy and safety of oral miglustat treatment in patients with mucopolysaccharidosis type III. The primary outcome was efficacy with improvement or stabilization in at least two domains of Vineland Adaptative Behavior Scales at 6 months. The secondary outcome measured the evolution of other cognitive tests at 12 months. The safety and tolerability were assessed throughout the study.Study designThis was a randomized, double-blind, placebo-controlled, monocenter, institutional, phase IIb to III study. In case of efficacy at 6 months, the study would go on for another 6 months on an open design with all patients receiving miglustat. In the absence of efficacy at 6 months, the trial had to be continued for 6 more months with the initial design.ResultsAfter 6 months, efficacy was not superior in patients with miglustat. The independent review board confirmed continuing the study until 12 months.ConclusionMiglustat treatment was not associated with any improvement/stabilization in behavior problems in patients with mucopolysaccharidosis type III. Miglustat has an acceptable safety profile. However, the study has confirmed that miglustat is able to pass through the blood-brain barrier without significantly decreasing ganglioside levels.

ناشر
Database: Elsevier - ScienceDirect (ساینس دایرکت)
Journal: The Journal of Pediatrics - Volume 159, Issue 5, November 2011, Pages 838-844.e1
نویسندگان
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