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Lysosomal Disorders

Gene Therapy Trial Makes Headway in Canavan Disease

Data from the first 3 of the 8 patients dosed indicates that the patients improved on the GMFM and the MSEL.

Gene Therapy Yields Improvements in Danon Disease

Rocket Pharma presented updated data from adult and pediatric patients at the 2022 HFSA meeting.

Advancing Neonatal Cell Therapy Research

Newborn Cell Therapies Group at Monash University has received a $50,000 grant from Lions Cord Blood foundation.