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 ...

- In a head-to-head comparison in a neuronopathic model for Gaucher disease, AL00804 exhibited higher potency and greater brain penetration compared to other GCS inhibitors currently in development - ...

NEW YORK, Dec. 26, 2019 (GLOBE NEWSWIRE) -- Prevail Therapeutics Inc. (Nasdaq: PRVL) (“Prevail” or the “Company”), a biotechnology company developing potentially disease-modifying AAV-based gene ...

NEW YORK, Oct. 27, 2020 (GLOBE NEWSWIRE) -- Prevail Therapeutics Inc. (Nasdaq: PRVL), a biotechnology company developing potentially disease-modifying AAV-based gene therapies for patients with ...

Gaucher disease, the inherited deficiency of lysosomal glucocerebrosidase, presents with a wide spectrum of manifestations. Although Gaucher disease has been divided into three clinical types, ...

BETHESDA, Md., Feb. 27, 2023 (GLOBE NEWSWIRE) -- Gain Therapeutics, Inc. (Nasdaq: GANX) (“Gain”, or the “Company”), a biotechnology company transforming drug discovery with its proprietary ...

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.