Gene therapy treatment in SMA with positive AAV9 antibodies

Case presentation: To describe the outcome of clinical evolution two SMA patients with positive test for AAV9 antibody, treated gene therapy. report: Patient 1, C.M.M., currently 2 years and 11 months, was diagnosed spinal muscular atrophy type C at 7 months due to loss cervical tonus reduction lower limbs movement, associated weight caused by dysphagia, initiation Spinraza treatment 10 months. At 33 he received therapy, a antibody (titer 1:100). The patient had no adverse events, only slight increase in transaminases, not higher than twice reference value. Only weeks after receiving it already possible observe effective cough improvement torso strength; After 45 days, able stand short orthosis. 2, T.E.S., 3 old, 17 age. He started nusinersena 19 25 an infusion therapy (Zolgensma) titer 1:100, while earlier 1:200. 1mg/kg/day prednisolone, without need dose. did present any major event, other transaminases up 4 times value, which allowed suspension corticosteroid 60 days infusion.