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Enzyme Replacement Therapy Equipment Supplied In Usa
9 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). ...
Manufactured by:BioMarin based inSan Rafael, CALIFORNIA (USA)
Vimizim (elosulfase alfa) is the first approved enzyme replacement therapy designed to address the underlying cause of Morquio A syndrome, or mucopolysaccharidosis IVA (MPS IVA) — a deficiency in the enzyme N-acetylgalactosamine-6 sulfatase (GALNS). VIMIZIM works at a cellular level to help with deficient ...
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 ...
Manufactured by:BioMarin based inSan Rafael, CALIFORNIA (USA)
Brineura (cerliponase alfa) is indicated to slow the loss of ambulation in symptomatic pediatric patients 3 years of age and older with late infantile neuronal ceroid lipofuscinosis type 2 (CLN2), also known as tripeptidyl peptidase 1 (TPP1) deficiency. Brineura is the first enzyme replacement therapy to be directly administered into the fluid of ...
Manufactured by:Entero Therapeutics, Inc. based inBoca Raton, FLORIDA (USA)
FW-EPI (adrulipase) is a recombinant lipase enzyme administered as an oral, non-systemic biologic capsule for the treatment of exocrine pancreatic insufficiency (EPI) associated with cystic fibrosis (CF) and chronic pancreatitis (CP). Adrulipase is derived from the Yarrowia lipolytica yeast lipase and is designed to break up fat molecules in the digestive tract of EPI patients so that they can be ...
by:Genova Diagnostics based inAsheville,, NORTH CAROLINA (USA)
A Non-Invasive Stool Biomarker of Pancreatic Exocrine Function. Pancreatic elastase is a protein-digesting enzyme exclusively produced by the human pancreas, and serves as a non-invasive stool biomarker of pancreatic exocrine function. Pancreatic elastase measured in stool has a strong correlation with the invasive gold-standard test for pancreatic insufficiency and, therefore, is a ...
Manufactured by:BioMarin based inSan Rafael, CALIFORNIA (USA)
ALDURAZYME (laronidase) is indicated for patients with Hurler and Hurler-Scheie forms of mucopolysaccharidosis I (MPS I) and for patients with the Scheie form who have moderate to severe symptoms. The risks and benefits of treating mildly affected patients with the Scheie form have not been established. ALDURAZYME has been shown to improve pulmonary function and walking capacity. ALDURAZYME has ...