- The P-I/II STRONG study involves assessing AVXS-101 (IT) in patients with SMA Type 2 who have three copies of the SMN2 gene, and who can sit but cannot stand or walk at the time of study entry aged ≥6 mos. but <2yrs. and patients who are ≥ 2yrs. but < 5yrs.
- The P-I/II study results: Patients aged ≥ 2yrs. & < 5yrs.) demonstrated improvement in Hammersmith Functional Motor Scale-Expanded (HFMSE) scores i.e, 5.9 point increase from baseline in HFMSE scores, at a mean duration of follow-up time of 9.3mos., half of the patients achieved ≥ 3-point increase starting at one-month post-treatment, presented at 24th WMS
- AVXS-101 (marketed as Zolgensma) is a gene therapy medication indicated for SMA, currently being evaluated in P-I/II study for IT administration in patients with SMA
Click here to read full press release/ article | Ref: Novartis | Image: Avexis