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P049: The long-chain fatty acid oxidation disorders (LC-FAOD) disease monitoring program: An evaluation of initial findings by Jerry Vockley, Barbara Burton, Amarilis Sanchez Valle, Michal Inbar-Feigenberg, Anjali Aggarwal, Joel Hetzer, Bridget Reineking, Andrew Grimm
Published 2025-01-01Get 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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