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 ...
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 ...
NEW YORK, Feb. 12, 2020 (GLOBE NEWSWIRE) -- Prevail Therapeutics Inc. (Nasdaq: PRVL) (“Prevail” or the “Company”), a biotechnology company developing potentially disease-modifying AAV-based gene ...
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 ...
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.
A Denali treatment for a rare lysosomal storage disorder, Hunter syndrome, has posted encouraging biomarker results in a small, open-label Phase 1/2 trial. The genetic disorder is caused by mutations ...
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