Intramuscular Administration of AAV1-Lipoprotein Lipase <sup>S447X</sup> Lowers Triglycerides in Lipoprotein Lipase–Deficient Patients
Erik S.G. Stroes(Amsterdam University Medical Centers), Jan Albert Kuivenhoven(University Medical Center Groningen), Colin J.D. Ross(BC Children's Hospital), Jaap Twisk(UniQure (Netherlands)), J. J. M. Meulenberg(Children's Hospital of Philadelphia), Michael R. Hayden(University of British Columbia), Mario Maas(Amsterdam UMC Location University of Amsterdam), Aeilko H. Zwinderman(Amsterdam Neuroscience), Katherine A. High(Spark Therapeutics (United States)), John J.P. Kastelein(QRx Pharma (United States)), Marcel M. Levi(University of Amsterdam), Remco Franssen(Children's Hospital of Philadelphia), Eleonora Aronica(Amsterdam Neuroscience), C. P. Henny, Melchior C. Nierman(Children's Hospital of Philadelphia)
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