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uniQure's pipeline is focused on (i) programs for orphan diseases with no effective treatment options (Porphyria, Sanfilippo B, LPLD) or
(ii) disruptive innovation programs destined to vastly improve the lot of patients and to change the dynamics of established markets (Parkinson’s disease, Hemophilia B)
(ii) disruptive innovation programs destined to vastly improve the lot of patients and to change the dynamics of established markets (Parkinson’s disease, Hemophilia B)