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As for LV-GCH1-TH-AADC, results of a phase I/II trial with two study sites (France and UK) showed long-term safety and tolerability of ProSavin , as well motor symptom improvement, as assessed with the UPDRS part 3 motor score

CappelliniMDBejaouiMAgaogluLCanatanDCapraMCohenAet alIron chelation with deferasirox in adult and pediatric patients with thalassemia major: efficacy and safety during 5 years follow-up