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

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P224: Clinical outcomes for patients enrolled in the MPS VII disease monitoring program (DMP) by Barbara Burton, Raymond Wang, Roberto Giugliani, Antonio Gonzalez-Meneses, Christina Grant, Maurizio Scarpa, Monica Penon Portmann, Esmeralda Oussoren, Esmeralda Martins, Consuelo Durand, Brigitte Chabrol, Joel Hetzer, Jeanne Cohen, J. Lawrence Merritt

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P325: The ClinGen Congenital Disorders of Glycosylation Gene Curation Expert Panel by Robin Larson, Raquel Fernandez, Carlos Ferreira, Hudson Freeze, Matthew Wilson, Dulce Quelhas, Jennifer Goldstein, Christina Lam, Bobby Ng, Heather Baudet, Daniel Beltran, Hongjie Chen, Andrew Edmondson, Miao He, Essence Kendall, Lois Lander, Jennifer McGlaughon, Eva Morava-Kosicz, Monica Penon Portmann, Jenny Thies, Meredith Weaver

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Mosaic X-linked adrenoleukodystrophy in males identified by newborn screening and next-generation sequencing by Alexandra C. Keefe, Dana M. Jensen, Meranda M. Pham, Natalie Y T Au, Erika Beckman, Monica Penon-Portmann, Emily Shelkowitz, Renee Bend, Michelle M. Morrow, Paul Kruszka, Divya Vats, Bianca E. Russell, Erica Chan, Derek Wong, Ahna Rabani, Lauren O’Grady, Inderneel Sahai, Kimberly Widmeyer, Ethan D. Sperry, Barbara E. Hallinan, Rebecca Tryon, Troy C. Lund, Florian S. Eichler, Angela Sun, James T. Bennett

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