Children with type I Gaucher disease: growing into adulthood with and without enzyme therapy

Prodromal substantia nigra sonography undermines suggested association between substrate accumulation and the risk for GBA-related Parkinson's disease.

European journal of neurology·2019

Management goals for type 1 Gaucher disease: An expert consensus document from the European working group on Gaucher disease.

Blood cells, molecules & diseases·2017

Treatment patterns from 647 patients with Gaucher disease: An analysis from the Gaucher Outcome Survey.

Blood cells, molecules & diseases·2016

Anthropometric adjustments are helpful in the interpretation of BMD and BMC Z-scores of pediatric patients with Prader-Willi syndrome.

Osteoporosis international : a journal established as result of cooperation between the European Foundation for Osteoporosis and the National Osteoporosis Foundation of the USA·2016

Taliglucerase alfa leads to favorable bone marrow responses in patients with type I Gaucher disease.

Blood cells, molecules & diseases·2012