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Efficacy and long-term safety of alipogene tiparvovec (AAV1-LPLS447X) gene therapy for lipoprotein lipase deficiency: an open-label trial.
Gaudet D, Méthot J, Déry S, Brisson D, Essiembre C, Tremblay G, Tremblay K, de Wal J, Twisk J, van den Bulk N, Sier-Ferreira V, van Deventer S. Gaudet D, et al. Among authors: sier ferreira v. Gene Ther. 2013 Apr;20(4):361-9. doi: 10.1038/gt.2012.43. Epub 2012 Jun 21. Gene Ther. 2013. PMID: 22717743 Free PMC article. Clinical Trial.