Sanfilippo syndrome, or mucopolysaccharidosis type III, is a neurological disease that attacks the brain and nervous system, causing children to gradually lose abilities such as language, learning, and motor skills. Type A is the most aggressive form, and most patients rarely live beyond their teenage years.
FDA clears 3.95 million dollar gene therapy
For the first time, there is now an approved gene therapy for Sanfilippo syndrome type A. The disease is extremely rare, and the treatment is among the most expensive in the world — with a list price of nearly four million dollars.
Advertisement
BREAKING