Dr. Kevin A. Strauss, Medical Director at the Clinic for Special Children, was an author on a recently published research paper, Clinical Trial and Postmarketing Safety of Onasemnogene Abeparvovec Therapy. This paper explores safety for onasemonogene abeparvovec therapy (gene therapy for Spinal Muscular Atrophy).
The study is the largest international safety study published thus far. Dr. Strauss contributed to the paper as the Clinic for Special Children served as a clinical site for the phase 3 SPR1NT study of the gene therapy. SMA is the most common genetic cause of infant death worldwide and causes progressive degeneration of motor nerve cells in the spinal cord and brainstem.
To read the full paper, please visit the link HERE.