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JR-441, Powder for solution for injection

JCR Pharmaceuticals Co., Ltd., JCR Pharmaceuticals Co., Ltd.

Mucopolysaccharidosis type IIIA (MPS IIIA)MPS III is an autosomal recessive disease characterized by the accumulation of HS in the tissues throughout the body due to a deficiency in enzymes involved in glycosaminoglycan (GAG) degradation in lysosomes. In MPSIII type A the deficient enzyme is SGSH. MPS IIIA has an estimated birth prevalence of between 0.26 and 1.89 per 100,000 live births and manifests as a severe neurodegenerative disease with progressive developmental regression., MPS IIIA is a condition that affects the brain. It can negatively impact how a person thinks and behaves. It can lead to difficulties with walking and sleeping, chest and ear infections, Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]