Dr. Yvan Torrente receives the "Neurologist of the Year 2021" award
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GFB INTERVIEW AT THE ITALIAN NATIONAL TELEVISION
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LGMD INTERNATIONAL CONFERENCE 2021 VIDEOS
Here the LGMD International conference 2021 videos
September 17 https://www.youtube.com/watch?v=Y2Ql5D1-gc8
The GFB VIdeo at 3 hour and 10 minutes
September 18 https://www.youtube.com/watch?v=XypFqwHfdyI
September 19 www.youtube.com/watch?v=XypFqwHfdyI&t=120s
September 20 www.youtube.com/watch?v=5KE4F7ohUAY
The GFB session at 46.30 minutes
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Sarepta Therapeutics’ Investigational Gene Therapy SRP-9003 for the Treatment of Limb-Girdle Muscular Dystrophy Type 2E Shows Sustained Expression and Functional Improvements 2 Years After Administration
3/18/21
-- Protein expression in muscle was sustained for two years following treatment in the low dose cohort, with mean beta-sarcoglycan expression of 54% at 24 months, compared to 36% at Day 60, as measured by western blot --
-- Mean NSAD score improvement of 5.7 points from baseline was sustained through 24 months in low-dose cohort, and mean NSAD score improvement of 4.0 points from baseline at one year in high-dose cohort --
-- Results in both cohorts continue to reinforce the safety and tolerability profile of SRP-9003 --
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