Disease characteristics, effectiveness, and safety of vestronidase alfa for the treatment of patients with mucopolysaccharidosis VII in a novel, longitudinal, multicenter disease monitoring program.

Autor: Giugliani R; Dep Genetics UFRGS, Casa dos Raros, INAGEMP, Med Genet Serv HCPA, and DASA Genomics, Porto Alegre, Brazil. rgiugliani@hcpa.edu.br., Gonzalez-Meneses A; Hospital Universitario Virgen del Rocio, Seville, Spain., Scarpa M; Regional Coordinator Centre for Rare Diseases, University Hospital of Udine, Udine, Italy., Burton B; Feinberg School of Medicine, Northwestern University, Chicago, IL, USA., Wang R; University of California Irvine School of Medicine, Children's Health of Orange County, Orange, CA, USA., Martins E; Centro Hospitalar Universitário de Santo António, Porto, Portugal., Oussoren E; Erasmus University Medical Center Rotterdam, Rotterdam, The Netherlands., Hennermann JB; University Medical Center Mainz, Villa Metabolica, Mainz, Germany., Chabrol B; Centre Hospitalier Universitaire La Timone, Marseille, France., Grant CL; Children's National Hospital, Washington, District of Columbia, USA., Sun A; Seattle Children's Hospital, Seattle, WA, USA., Durand C; Laboratorio Chamoles, Buenos Aires, Argentina., Hetzer J; Ultragenyx Pharmaceutical Inc, Novato, CA, USA., Malkus B; Ultragenyx Pharmaceutical Inc, Novato, CA, USA., Marsden D; Ultragenyx Pharmaceutical Inc, Novato, CA, USA., Merritt Ii JL; Ultragenyx Pharmaceutical Inc, Novato, CA, USA.

Publikováno v: Orphanet journal of rare diseases [Orphanet J Rare Dis] 2024 May 07; Vol. 19 (1), pp. 189. Date of Electronic Publication: 2024 May 07.