Sanfilippo Syndrome Market is driven by Gene Therapy Advances

added blog Health

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...

0 Comments 0 Shares 226 Views 0 Reviews