First-in-human Phase 1/2 trial of intravenous FBX-101 with Cord Blood Transplantation increases GALC, brain and motor development in Infantile Krabbe
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Presenting Author - Maria L. Escolar, MD, MS; Co-Author - Mark Vander Lugt, MD; Co-Author - Michele Poe, PhD; Co-Author - Melissa Greco, BS; Co-Author - Keith Werling, PhD; Co-Author - Erandi K. De Silva, PhD; Co-Author - Juan Ruiz, MD, PhD, MBA; Co-Author - Paul Szabolcs, MD;
Meta Tag
Brain/Nervous System
Lysosomal Diseases
Therapy
Transplantation
Co-Author Mark Vander Lugt, MD
Co-Author Michele Poe, PhD
Co-Author Melissa Greco, BS
Co-Author Keith Werling, PhD
Co-Author Erandi K. De Silva, PhD
Co-Author Juan Ruiz, MD, PhD, MBA
Co-Author Paul Szabolcs, MD
Presenting Author Maria L. Escolar, MD, MS
Keywords
FBX-101
gene therapy
infantile Krabbe disease
GALC gene
safety
efficacy
motor development
RESKUE trial
hematopoietic stem cell transplantation
newborn screening

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