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Gain Enzyme Replacement Therapy 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 and central nervous system (CNS). ...
by:Gain Therapeutics, Inc. based inBethesda, MARYLAND (USA)
Mucopolysaccharidosis type 1 (MPS 1) is a rare lysosomal storage disease that is caused by mutations in IDUA, the gene that encodes the alpha-L-iduronidase (IDUA) enzyme. These mutations cause the misfolding and dysfunction of IDUA, which leads to the toxic buildup of large sugars in the bone, cartilage, cornea, heart and central nervous system (CNS). There is currently no cure ...
by:Gain Therapeutics, Inc. based inBethesda, MARYLAND (USA)
Morquio B, also known as Mucopolysaccharidosis type IV (MPS IV), is a progressive disease mostly impacting the skeleton, caused by mutations in GLB1, the gene that encodes the beta-galactosidase (GLB) enzyme. These mutations result in the misfolding and subsequent dysfunction of GLB, which leads to the toxic substrate accumulation of keratan sulfate in organs and tissues. Very ...
by:Gain Therapeutics, Inc. based inBethesda, MARYLAND (USA)
GM1 Gangliosidosis is a hereditary, progressive disease mostly impacting neurons in the brain and spinal cord, caused by mutations in GLB1, the gene that encodes the beta-galactosidase (GLB) enzyme. These mutations result in the misfolding and subsequent dysfunction of GLB, which leads to the toxic substrate accumulation of GM1 ganglioside in organs and tissues. Very limited and ...