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 ...
WebMD

What to Know About Avlayah for Hunter Syndrome

Hunter syndrome, also known as mucopolysaccharidosis II (MPS II), is a rare inherited condition that mainly affects boys. It causes certain substances to build up in the body, leading to damage to ...
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.
manilatimes

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 ...
FierceBiotech

FDA details rationale for rejecting rare disease gene therapy from Regenxbio

The FDA has released the rejection letter explaining its recent refusal of Regenxbio’s gene therapy for the rare disease Hunter syndrome, providing further details into the agency’s issues with the ...