Study of AAVrh10-h.SGSH Gene Therapy in Patients With Mucopolysaccharidosis Type IIIA (MPS IIIA)
Status:
Active, not recruiting
Trial end date:
2022-03-01
Target enrollment:
Participant gender:
Summary
MPS IIIA is predominantly a central nervous system disease causing cognitive disability,
progressive loss of acquired skills, behavioral and sleep disturbance. LYS-SAF302 is a gene
therapy which is intended to deliver a functional copy of the SGSH gene to the brain. This is
a phase 2-3 study to assess the efficacy in improving or stabilizing the neurodevelopmental
state of MPS IIIA patients.