Efficacy and long-term safety of alipogene tiparvovec (AAV1-LPLS447X) gene therapy for lipoprotein lipase deficiency: an open-label trial
Daniel Gaudet(Brigham and Women's Hospital), S van Deventer(AIMM Therapeutics (Netherlands)), Valerie Sier-Ferreira(AIMM Therapeutics (Netherlands)), C Essiembre(Université du Québec à Chicoutimi), S. Déry(Université du Québec à Chicoutimi), Julie Méthot(Université du Québec à Chicoutimi), Karine Tremblay(Université de Montréal), Diane Brisson(Université de Montréal), N. van den Bulk(AIMM Therapeutics (Netherlands)), G Tremblay(Université du Québec à Chicoutimi), Jaap Twisk(UniQure (Netherlands)), J. de Wal(AIMM Therapeutics (Netherlands))
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