P189: Vestronidase alfa for the treatment of mucopolysaccharidosis VII (MPS VII): Updated results from a novel, longitudinal, multicenter disease monitoring program
Roberto Giugliani,
Antonio Gonzalez-Meneses,
Christina Grant,
Maurizio Scarpa,
Angela Sun,
Raymond Wang,
Joel Hetzer,
Deborah Marsden,
J. Lawrence Merritt, II
Affiliations
Roberto Giugliani
Department of Genetics UFRGS, Medical Genetics Services HCPA, and INAGEMP, Porto Alegre, Brazil
Antonio Gonzalez-Meneses
Hospital Universitario Virgen del Rocio, Seville, Spain
Christina Grant
Children’s National Hospital, Washington, DC
Maurizio Scarpa
Regional Coordinator Centre for Rare Diseases, University Hospital of Udine, Udine, Italy
Angela Sun
Seattle Children’s Hospital, Seattle, WA
Raymond Wang
Children’s Hospital of Orange County and University of California-Irvine, Orange, CA
