Disease characteristics, effectiveness, and safety of vestronidase alfa for the treatment of patients with mucopolysaccharidosis VII in a novel, longitudinal, multicenter disease monitoring program
- Roberto Giugliani*
- , Antonio Gonzalez-Meneses
- , Maurizio Scarpa
- , Barbara Burton
- , Raymond Wang
- , Esmeralda Martins
- , Esmeralda Oussoren
- , Julia B. Hennermann
- , Brigitte Chabrol
- , Christina L. Grant
- , Angela Sun
- , Consuelo Durand
- , Joel Hetzer
- , Betsy Malkus
- , Deborah Marsden
- , J. Lawrence Merritt
*Corresponding author for this work
- Universidade Federal do Rio Grande do Sul
- Hospital Universitario Virgen del Rocio
- University of Udine
- Northwestern University
- University of California at Irvine
- University Hospital Center of Santo António
- Johannes Gutenberg University Mainz
- CHU Timone
- Children’s National Hospital
- Seattle Children's Hospital
- Laboratorio Chamoles
- Ultragenyx Pharmaceutical Inc.
Research output: Contribution to journal › Article › Academic › peer-review
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