Sio Gene Therapies Announces CSF Reductions in GM1 Ganglioside from Clinical Trial of AXO-AAV-GM1 Gene Therapy streetinsider.com - get the latest breaking news, showbiz & celebrity photos, sport news & rumours, viral videos and top stories from streetinsider.com Daily Mail and Mail on Sunday newspapers.
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Sio Gene Therapies Announces Positive Six-Month Follow-Up Data from Low-Dose Cohort of Phase .
Sio Gene TherapiesDecember 15, 2020 GMT
- Generally well-tolerated with a favorable safety profile in five patients
- Serum beta-galactosidase enzyme activity increased in all patients at all timepoints between Day 7 and Month 6, representing an approximate doubling in enzyme activity after gene transfer
- At Month 6, enzyme activity restored to 23-57% (mean: 38%) of normal reference levels
- All five children demonstrated signs of clinical disease stability as assessed by Vineland-3 Growth Scale Value, Upright and Floor Mobility, and Clinical Global Impression (CGI) scales