The trial is expected to include 14 individuals with genetically confirmed limb-girdle muscular dystrophy who will be randomly assigned 1:1 to either AB-1003 or placebo for a year-long treatment period.
In a dose-ranging study, the gene therapy achieved 78% Gb3 substrate clearance at 6 months and 77% reduction in urine podocyte loss in 1 of the first kidney biopsies.
To honor Black History Month, NeurologyLive® spoke with influential Black clinicians on the leaders they look up to, the ongoing fight to overcome racial disparities, and ways to encourage diversity in health care.
To honor Black History Month, NeurologyLive® spoke with influential Black clinicians on the leaders they look up to, the ongoing fight to overcome racial disparities, and ways to encourage diversity in health care.
To honor Black History Month, NeurologyLive® spoke with influential Black clinicians on the leaders they look up to, the ongoing fight to overcome racial disparities, and ways to encourage diversity in health care.