Risultati della ricerca - Maria Escolar

O30: REKLAIM: A first in human phase Ib clinical trial of FBX-101 (AAVrh10.GALC) intravenously administered after UCBT for infantile Krabbe disease di Maria Escolar, Mark Vander Lugt, Raymond Wang, Juan Ruiz, Michele Poe, Paul Szabolcs

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Infarto humeral diafisario en paciente con anemia falciforme di Einés Monteagudo Vilavedra, Manuel Emilio García Fernández, José María Escolar Martín, Andrea Sánchez Domínguez

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P014: First-in-human phase 1/2 trial of intravenous FBX-101 with cord blood transplantation increases GALC, brain and motor development in infantile Krabbe di Maria Escolar, Mark Vander Lugt, Michele Poe, Melissa Greco, Keith Werling, Erandi De Silva, Juan Ruiz, Paul Szabolcs

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Fluorimetric assay with a novel substrate for quantification of galactocerebrosidase activity in dried blood spot specimens di Anirudh J. Ullal, Hong Pham, Rajendra Singh, Peter Ross, Carrie A. Graham, Scott M. Norton, Miriam H. Nuffer, Debbie S. Burns, Allen E. Eckhardt, Maria Escolar, Deeksha Bali, Vamsee K. Pamula

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Development of a Clinical Algorithm for the Early Diagnosis of Mucopolysaccharidosis III di Maria Escolar, Jessica Bradshaw, Valerie Tharp Byers, Roberto Giugliani, Lynn Golightly, Charles Marques Lourenço, Kimberly McDonald, Nicole Muschol, Imogen Newsom-Davis, Cara O’Neill, Holly L. Peay, Jennifer Siedman, Martha L. Solano, Tessa Wirt, Tim Wood, Lonnie Zwaigenbaum

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Population-Based Screening of Newborns: Findings From the NBS Expansion Study (Part One) di Amy Brower, Kee Chan, Marc Williams, Susan Berry, Robert Currier, Piero Rinaldo, Michele Caggana, Amy Gaviglio, William Wilcox, Robert Steiner, Ingrid A. Holm, Jennifer Taylor, Joseph J. Orsini, Luca Brunelli, Joanne Adelberg, Olaf Bodamer, Sarah Viall, Curt Scharfe, Melissa Wasserstein, Jin Y. Chen, Maria Escolar, Aaron Goldenberg, Kathryn Swoboda, Can Ficicioglu, Dieter Matern, Rachel Lee, Michael Watson

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