P261: Vestronidase alfa for the treatment of mucopolysaccharidosis VII (MPS VII): Updated results from a longitudinal, multicenter disease monitoring program (DMP)

• Roberto Giugliani,
• Antonio Gonzalez-Meneses,
• Christina Grant,
• Maurizio Scarpa,
• Angela Sun,
• Raymond Wang,
• Barbara Burton,
• Ans van der Ploeg,
• Esmeralda Martins,
• Consuelo Durand,
• Brigitte Chabrol,
• Joel Hetzer,
• Deborah Marsden,
• J. Lawrence Merritt, II

Affiliations

Roberto Giugliani

Dep Genetics UFRGS, Med Genet Serv HCPA, INAGEMP, DASA and Casa dos Raros, 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

Barbara Burton

Ann & Robert H. Lurie Children's Hospital of Chicago, Chicago, IL

Ans van der Ploeg

Erasmus University Medical Center Rotterdam, Rotterdam, The Netherlands

Esmeralda Martins

Centro Hospitalar do Porto, Porto, Portugal

Consuelo Durand

Laboratorio Chamoles, Buenos Aires, Argentina

Brigitte Chabrol

Centre Hospitalier Universitaire La Timone, Marseille, France

Joel Hetzer

Ultragenyx Pharmaceutical Inc

Deborah Marsden

Ultragenyx Pharmaceutical Inc

J. Lawrence Merritt, II

Ultragenyx Pharmaceutical Inc