Nature

Characterization of neuronopathic Gaucher disease among ethnic Poles

Purpose: Gaucher disease is a common lysosomal storage disease that results from inherited mutations in the gene (GBA) encoding acid β-glucosidase (GCase). Here, the clinical and molecular findings of ...
Nature

An increase in ER stress and unfolded protein response in iPSCs-derived neuronal cells from neuronopathic Gaucher disease patients

Gaucher disease (GD) is a lysosomal storage disorder caused by a mutation in the GBA1 gene, responsible for encoding the enzyme Glucocerebrosidase (GCase). Although neuronal death and ...

Constantiam Biosciences and Cincinnati Children’s Collaborate to Accelerate First-in-Class Neuronopathic Gaucher Disease Therapies

WATERTOWN, Mass. and CINCINNATI, June 18, 2026 (GLOBE NEWSWIRE) -- Constantiam Biosciences and Cincinnati Children's today announced a strategic collaboration through an exclusive option for future ...
Business Wire

AVROBIO Announces Neuronopathic Mucopolysaccharidosis Type II (nMPS-II) or Hunter Syndrome Clinical Trial Application (CTA) Accepted by U.K. Regulatory Agency to Initiate ...

CAMBRIDGE, Mass.--(BUSINESS WIRE)--AVROBIO, Inc. (Nasdaq: AVRO), a leading clinical-stage gene therapy company working to free people from a lifetime of genetic disease, today announced that the U.K.
Business Wire

The International Gaucher Alliance and Kantar Health Launch the GARDIAN Registry to Accelerate the Understanding of Neuronopathic Gaucher Disease

NEW YORK--(BUSINESS WIRE)--The International Gaucher Alliance (IGA) and Kantar Health announce today the launch of the disease registry GARDIAN: Gaucher Registry for Development Innovation and ...