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, Oct. 27, 2020 (GLOBE NEWSWIRE) -- Prevail Therapeutics Inc. (Nasdaq: PRVL), a biotechnology company developing potentially disease-modifying AAV-based gene therapies for patients with ...
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 ...
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.
Centogene NV, the essential life science partner for data-driven answers in rare and neurodegenerative diseases, announced the discovery of a new small molecule together with Evotec SE. The new ...
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 ...
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 ...
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, ...
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