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Unmet needs of adults living with mucopolysaccharidosis II: data from the Hunter Outcome Survey by Joseph Muenzer, Hernan Amartino, Roberto Giugliani, Paul Harmatz, Shuan-Pei Lin, Bianca Link, David Molter, Uma Ramaswami, Maurizio Scarpa, Jaco Botha, Jennifer Audi, Barbara K. Burton
Published 2025-07-01Get full text
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P026: Interim analysis of the efficacy and safety of weekly intravenous tividenofusp alfa in mucopolysaccharidosis type II: A phase 1/2 study by Barbara Burton, Joseph Muenzer, Paul Harmatz, Deepa Rajan, Simon Jones, Johanna van den Hout, John Mitchell, Matthew Troyer, Natalie Engmann, Rupa Caprihan, Akhil Bhalla, Imanol Zubizarreta, Arlette Weichert, Peter Chin, Carole Ho
Published 2025-01-01Get full text
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