Dose selection for intracerebroventricular cerliponase alfa in children with CLN2 disease, translation from animal to human in a rare genetic disease
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Published in Clinical and translational science (01.09.2021)
Published in Clinical and translational science (01.09.2021)
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Clinical Pharmacokinetics and Pharmacodynamics of Cerliponase Alfa, Enzyme Replacement Therapy for CLN2 Disease by Intracerebroventricular Administration
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Published in Clinical and translational science (01.03.2021)
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Vector Shedding and Blood Biodistribution in Patients with Severe Hemophilia a Following Administration of Valoctocogene Roxaparvovec
Clark, Annie, Hammon, Kevin, Sandza, Krystal, Torres, Richard, Koziol, Elli, Holcomb, Jennifer, Kim, Benjamin, Jayaram, Kala, Russell, Chris, Vettermann, Christian, Henshaw, Joshua
Published in Blood (05.11.2020)
Published in Blood (05.11.2020)
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Sandza, Krystal, Clark, Annie, Koziol, Elli, Akeefe, Hassibullah, Yang, Fan, Holcomb, Jennifer, Patton, Kathryn, Hammon, Kevin, Mitchell, Nina, Wong, Wing Y, Zoog, Stephen J, Kim, Benjamin, Henshaw, Joshua, Vettermann, Christian
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Published in Gene therapy (01.02.2022)
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