The lysosomal storage diseases (LSDs) are a group of inherited metabolic disorders that are
caused for the most part by enzyme deficiencies within the lysosome resulting in …

Gaucher disease, the most common lysosomal storage disorder, is caused by the defective
activity of the lysosomal enzyme, acid‐β‐glucosidase (GlcCerase), leading to accumulation …

Gaucher disease (GD) encompasses a continuum of clinical findings from a perinatal lethal
disorder to an asymptomatic type. The identification of three major clinical types (1, 2, and 3) …

Study of the natural history of Gaucher disease has revealed marked phenotypic variation.
Correlations to genotypes could provide insight into individual susceptibility to varying …

Gaucher disease (GD) is an autosomal recessive inherited lysosomal storage disease that
often presents in early childhood and is associated with damage to multiple organ systems …

Objective To describe the clinical and demographic characteristics of nonneuronopathic
Gaucher disease (GD) in children at the time of diagnosis. Design Longitudinal …

A global, observational disease registry has been established to characterize the course of
disease and track clinical outcomes in patients with Mucopolysaccharidosis Type I (MPS I), a …

Gaucher disease is an inherited pan-ethnic disorder that commonly begins in childhood and
is caused by deficient activity of the lysosomal enzyme glucocerebrosidase. Two major …

Gaucher disease is a rare multi-systemic metabolic disorder caused by the inherited
deficiency of the lysosomal enzyme β-glucocerebrosidase, which leads to the accumulation …

5,622,940 A 4, 1997 Ostroff The present invention provides a particulate delivery system
5,633,369 A 5, 1997 Jamas et al. comprising an extracted yeast cell wall comprising beta …