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Een enorme mijlpaal voor uniQure nu in de VS de eerste twee patiënten met de ziekte van Huntington zijn geïnjecteerd. Doel is met een geïnjecteerd virus in de hersenen de aanmaak van het Huntington eiwit remmen. Het is een phase I/II trial wat officieel voor het onderzoeken van de veiligheid ervan is (en bijwerkingen) maar het is ook meteen 'the real deal' want de aanwezige ASO gaat aan het werk. Dus ja een echte 'Milestone' voor dit Nederlandse bedrijf!


uniQure treats first two patients in Phase I/II trial of AMT-130 uniQure announced that the first two patients in the Phase I/II clinical trial of AMT-130 for the treatment of Huntington's disease have been treated. The Phase I/II study is a double-blind, randomized clinical trial being conducted in the United States, with now one patient treated with AMT-130, and one patient who received the imitation surgery. The Phase I/II clinical trial of AMT-130 for the treatment of Huntington's disease will explore the safety, tolerability, and efficacy signals in 26 patients with early manifest Huntington's disease randomized to treatment with AMT-130 or an imitation surgery. The five-year, multi-center trial consists of a blinded 18-month core study period followed by unblinded long-term follow-up. Patients will receive a single administration of AMT-130 through MRI-guided, convection-enhanced stereotactic neurosurgical delivery directly into the striatum. The first two patients will be observed for an initial period of 90 days, followed by a meeting of the Data Safety Monitoring Board. The DSMB will review the data on the first two patients and make a determination about continued dosing of the next patients. AMT-130 is uniQure's first clinical program focusing on the central nervous system incorporating its proprietary miQURE platform.