The P-II/III (AAVance) trial evaluate LYS-SAF302 in 20 patients with MPS IIIA. The full study results are expected in mid-Sept. 2022, along with results from the (PROVide) PROs videos study
The positive biomarker data was confirmed in additional subjects & at additional time points with a significant reduction of about 20% on avg. levels of total HS-derived oligosaccharides in the CSF relative to baseline levels at 6, 12, and 24mos., HS levels were reduced by 22% @24mos. with no significant effect on serum HS levels at 6, 12, or 24mos.
Stable or continuously increasing cognitive, language & motor functions in 3 patients treated at ≤31mos. of age incl. patients with SGSH mutations associated with the severe disease phenotype
Ref: Bussinesswire | Image: Lysogene
