SGT-001 is currently being evaluated in a phase 1/2 study called IGNITE DMD (NCT03368742).91Interim analysis of 2 years (n = 9 boys) demonstrated common drug-related laboratory abnormalities (thrombocytopenia, anemia), and serious adverse events: systemic inflammatory response syndrome (n = 2), thrombocytopenia (n = 1), and immune hepatitis (n = 1), which were resolved.91Results showed durable microdystrophin expression and localization on nNOS to the membrane in biopsies collected at time points ranging from 12-24 months post-dosing. This evidence concerns the gene NOS1 and Thrombocytopenia.