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SGT-001 is a novel adeno-associated viral (AAV) vector-mediated gene transfer therapy for Duchenne muscular dystrophy (DMD).
The data will be presented today at the World Muscle Society 2021 Virtual Congress.
The data demonstrated durable expression and function of microdystrophin protein after 12 to 24 months post-dosing.
NSAA scores (measurement of functional motor abilities) showed minimal change compared with baseline and suggested benefit after treatment compared to trajectories typically observed in natural history data.
Natural history analyses suggest that patients would usually exhibit a 4.5-point decline in NSAA over 1.5 years.
In contrast, patients exhibited a mean decrease of 1.7 points from baseline and a mean difference of +2.8 points compared with natural history data.
6-Minute Walk Test (6MWT) distances were maintained 1.5 years post-dosing, while natural history analyses suggested a 63.5-meter decline.
The mean increase in the 6MWT was 15.3 meters compared with baseline, and the mean difference compared with natural history data was +78.8 meters.
The percent predicted FVC (lung function tests) continued to show stability or improvement, while natural history analyses suggest a decline of 7.5%.
Patient-reported outcome measures showed meaningful, sustained improvements at 1.5 years compared with baseline.
No new drug-related safety findings were reported.
Price Action: SLDB stock is up 1% at $3.03 during the market session on the last check Thursday.
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