Breakthrough in Genetic Disorder Treatment: GC Pharma’s Sanfilippo Syndrome Drug Receives FDA Phase 1 Approval
Sanfilippo Syndrome, also known as Mucopolysaccharidosis Type III, is a rare genetic disorder caused by the body’s inability to produce an enzyme necessary for breaking down heparan sulfate. This accumulation leads to severe organ damage and developmental delays. GC Pharma, in collaboration with biotech firm Nobel Pharma, has announced that their innovative treatment for Sanfilippo … 더 읽기
