Enzyme replacement therapy with galsulfase for mucopolysaccharidosis VI: clinical facts and figures by Paul Harmatz

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Current and new therapies for mucopolysaccharidoses by Monica Penon-Portmann, David R. Blair, Paul Harmatz

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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

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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

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O13: Oral infigratinib for children with achondroplasia: Month 18 results from the PROPEL 2 study by Ravi Savarirayan, Elena Muslimova, Josep Bergua, Paul Arundel, Jean Salles, Vrinda Saraff, Borja Delgado, Antonio Leiva-Gea, Helen McDevitt, Marc Nicolino, Massimiliano Rossi, Maria Salcedo, Valerie Cormier-Daire, Mars Skael, Peter Kannu, John Phillips, Howard Saal, Paul Harmatz, Toby Candler, Richard Weng, Yun Bai, Supriya Raj, Julie Hoover-Fong, Melita Irving, Daniela Rogoff

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