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Phenotype differentiation and white matter preservation by early hematopoietic cell transplantation - Free-water diffusion tensor MRI study of the brain in mucopolysaccharidosis type I patients Nestrasil, Igor; Svatkova, Alena; Pasternak, Ofer; Eisengart, Julie B.; Rudser, Kyle; Mueller, Bryon A.; Bednarik, Petr; Delaney, Kate; Shapiro, Elsa; Whitley, Chester B. Share Save
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Outcome of hematopoietic stem cell transplantation in 19 patients with alpha-mannosidosis Saho, Robert; Formankova, Renata; Eisengart, Julie B.; Lund, Allan; Videbaek, Cecilie; Gurbuz, Berrak Bilginer; Ozbek, Namik Yasar; Al Jasmi, Fatma; Jesina, Pavel; Feillet, Francois; Pochon, Cecile; Guemann, Anne-Sophie; Alsayed, Moeen; Laktina, Sabine; Ucar, Sema Kalkan; Aksoylar, Serap; Lund, Troy; Orchard, Paul J.; Eminoglu, Fatma T.; Ileri, Talia; Kasapkara, cigdem S.; Yesilipek, Akif; Tuncel, Ali Tunc; Jurickova, Katarina; Magner, Martin Share Save
Bone marrow transplantation reverses metabolic alterations in multiple sulfatase deficiency: a case series Pillai, Nishitha R.; Liu, Ning; Li, Xiyuan; Li, Xiqi; Ahrens-Nicklas, Rebecca; Adang, Laura; Eisengart, Julie B.; Bronken, Grace; Gupta, Ashish; Lund, Troy C.; Whitley, Chester B.; Elsea, Sarah H.; Orchard, Paul J. Share Save
Anakinra in Sanfilippo syndrome: a phase 1/2 trial (Jul, 10.1038/s41591-024-03079-3, 2024) Polgreen, Lynda E.; Chen, Agnes H.; Pak, Youngju; Luzzi, Anna; Garval, Adolfo Morales; Acevedo, Jonathan; Bitan, Gal; Iacovino, Michelina; O'Neill, Cara; Eisengart, Julie B. Share Save
Anakinra in Sanfilippo syndrome: a phase 1/2 trial Polgreen, Lynda E.; Chen, Agnes H.; Pak, Youngju; Luzzi, Anna; Garval, Adolfo Morales; Acevedo, Jonathan; Bitan, Gal; Iacovino, Michelina; O'Neill, Cara; Eisengart, Julie B. Share Save
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New blood-based biomarkers for Sanfilippo syndrome Bitan, Gal; Taha, Hash Brown; Siddique, Ibrar; Chamoun, Lea M.; Mohseni, Kimia; Morris, Alanna J.; Luzzi, Anna; Acevedo, Jonathan; Morales, Adolfo; de la Garza, Giselle Perez; Nour, Elabed; Khijniak, Marie; Fenwick, Leony; Chen, Agnes; Iacovino, Michelina; O'Neill, Cara; Eisengart, Julie B.; Polgreen, Lynda Share Save
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Enzyme replacement is associated with better long-term cognitive outcomes in Hurler syndrome Greenberg, Miles C.; Orchard, Paul J.; Gupta, Ashish; Braunlin, Elizabeth; Miller, Bradley; Pillai, Nishitha R.; King, Kelly; Whitley, Chester B.; Lund, Troy; Eisengart, Julie B. Share Save
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