First-in-human phase 1/2 trial of intravenous FBX-101 following hematopoietic stem cell transplantation increases GALC activity, supports brain development, and improves motor function in patients with infantile Krabbe disease: RESKUE clinical trial

Escolar, Maria L.; Lugt, Mark Vander; Poe, Michele D.; Greco, Melissa; Werling, Keith; Silva, Erandi De; Ruiz, Juan; Szabolcs, Paul

doi:10.1016/j.ymgme.2022.107091

Molecular Genetics and Metabolism

2023

DOI: 10.1016/j.ymgme.2022.107091

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First-in-human phase 1/2 trial of intravenous FBX-101 following hematopoietic stem cell transplantation increases GALC activity, supports brain development, and improves motor function in patients with infantile Krabbe disease: RESKUE clinical trial

Maria L. Escolar¹,

Mark Vander Lugt²,

Michele D. Poe³

et al.

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First-in-human phase 1/2 trial of intravenous FBX-101 following hematopoietic stem cell transplantation increases GALC activity, supports brain development, and improves motor function in patients with infantile Krabbe disease: RESKUE clinical trial

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