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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...

Emerging Therapeutics in Preeclampsia Management

Preeclampsia is a pregnancy related complication that affects over 5% to 8% of pregnancies or about 3,00,000 women in the US alone. An estimated 10,500 babies in the U.S, succumb to preeclampsia while others suffer from extensive damage owing to premature delivery. It is one of...

Is Autologous Stem Cell Therapy the next frontier of medicine?

Stem Cell Therapy is emerging as the future of therapeutics for numerous diseases and disorders which have hitherto been unaddressed. The growing awareness of the regenerative powers of stem cells, along with rising research and development expenditure on stem cell technology will drive the growth...

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