Gain - Mucopolysaccharidosis Type 1 (Mps 1)
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 for these patients; however, enzyme replacement therapy and hematopoietic stem cell transplant are used to manage the non-CNS symptoms. Gain is developing allosteric regulators to increase IDUA activity and decrease toxic sugar accumulation in the CNS of patients with MPS 1 to potentially become the first treatment for the disease’s neuronopathic symptoms.
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