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Gaucher Disease Equipment & Supplies
4 equipment items found
by:Gain Therapeutics, Inc. based inBethesda, MARYLAND (USA)
Gaucher disease is the most common lysosomal storage disease and is caused by mutations in GBA, the gene that encodes the beta-glucocerebrosidase (GCase) enzyme. These mutations result in the misfolding and subsequent dysfunction of GCase, which leads to the toxic buildup of fat in a variety of organs and tissues such as the liver, spleen, bones ...
Manufactured by:Tectonic Therapeutic, Inc. based in
AVROBIO’s investigational gene therapy for Gaucher disease is being studied in a Phase 1/2 clinical trial to evaluate the safety and efficacy in individuals with Gaucher disease type 1. The trial is now enrolling in Canada, Australia and the U.S. The trial is intended to recruit 8 to 16 individuals between the ages of 16 ...
Manufactured by:Tectonic Therapeutic, Inc. based in
AVROBIO has a preclinical research program for an investigational gene therapy for Gaucher disease type ...
by:LIXTE Biotechnology Holdings, Inc. based inPasadena, CALIFORNIA (USA)
Designated as our LB-200 series of compounds, these have potential use in the prevention and treatment of neurodegenerative diseases. The series has the potential to be the most effective in its class and may be useful for the treatment of chronic hereditary diseases, such as Gaucher’s disease, in addition to cancer and ...