Sanfilippo Syndrome Market is driven by Gene Therapy Advances

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2025-05-21 11:49:53 -

Sanfilippo syndrome, also known as mucopolysaccharidosis type III, is a rare genetic disorder characterized by the body’s inability to break down heparan sulfate. Treatments in development include enzyme replacement therapies, substrate reduction therapies, gene therapies, and small-molecule drugs that target lysosomal storage pathways. These products offer advantages such as improved...

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