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5 Companies unraveling the potential of Gene Based Therapies for the treatment of Sanfilippo syndrome (Mucopolysaccharidosis type III (MPS III)

Mucopolysaccharidosis type III (MPS III), also known as Sanfilippo syndrome, is caused by the mutations in the glucosamine (N-acetyl)-6-sulfatase (GNS), heparan-alpha-glucosaminide N-acetyltransferase (HGSNAT), N-acetyl-alpha-glucosaminidase (NAGLU) and N-sulfoglucosamine sulfohydrolase (SGSH) genes, which are responsible for the synthesis of large sugar molecules called glycosaminoglycans (GAGs). The dysfunction...

Top 5 Emerging Therapeutics for Crigler – Najjar Syndrome

Crigler Najjar Syndrome, also called hyperbilirubinemia, is a chronic condition characterized by high levels of unconjugated bilirubin in the blood. The disease manifests as Jaundice at birth, and can progressively lead to kernicterus, a condition in which the accumulation of unconjugated bilirubin can damage brain...

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