New data from the AAVance trial has revealed children with Sanfilippo syndrome type A, including those with severe disease, showed stable or continuously increasing cognitive, language, and motor functions two years after dosing with the investigational gene therapy LYS-SAF302. The therapy also maintained low levels of the disease-related biomarker heparan sulfate…
MPS III Gene Therapy LYS-SAF302 Boosts or Stabilizes Development After 2 Years
