Title : GDF15 as a new useful biomarker of mitochondrial disorders, and develop the diagnostic device in Japan
Abstract:
Among pediatricians and neurologists who treat patients with MELAS, there is an urgent need for clinical evidence on the effective treatment for them. To date, pharmacotherapy with L-arginine has been suggested to be promising for patients with MELAS. We conducted 9-year clinical research with MELAS, into which the data from two 2-year, phase 3, multicenter, open-label clinical trials of oral and intravenous L-arginine involving 7-year follow-up were integrated as pooled data. We will discuss the therapeutic regimen in the presentation.