Gene Therapy Benefits Children with Sanfilippo Syndrome, Phase 1/2 Trial Shows
A gene therapy led to the brain producing the proper form of an enzyme whose faulty version causes Sanfilippo syndrome type B, a Phase 1/2 clinical trial showed. The therapy, rAVV2/5, also improved the neurological condition and behavior of the four preschool-age children involved in the study (ISRCTN19853672), researchers said.