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Cyclerion Therapeutics Praliciguat Fails in Two Crucial Clinical Trials

Cyclerion Therapeutics (NASDAQ: CYCN), a clinical-stage Company, announced the failure of its drug candidate praliciguat in meeting the primary endpoints in two separate clinical studies, in diabetic nephropathy (DN) and heart failure with preserved ejection fraction (HFpEF). Praliciguat is a soluble guanylate cyclase (sGC) stimulator...

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...

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