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Use of the Bruininks-Oseretsky test of motor proficiency (BOT-2) to assess efficacy of velmanase alfa as enzyme therapy for alpha-mannosidosis.
Phillips, Dawn; Hennermann, Julia B; Tylki-Szymanska, Anna; Borgwardt, Line; Gil-Campos, Mercedes; Guffon, Nathalie; Amraoui, Yasmina; Geraci, Silvia; Ardigò, Diego; Cattaneo, Federica; Lund, Allan M.
Afiliación
  • Phillips D; UNC Chapel Hill, Division of Physical Therapy, School of Medicine, Chapel Hill, NC, United States of America.
  • Hennermann JB; University Medical Centre Mainz, Dept. Pediatric and Adolescent Medicine, Villa Metabolica, Mainz, Germany.
  • Tylki-Szymanska A; Department of Paediatric, Nutrition and Metabolic Diseases, The Children's Memorial Health Institute, Warsaw, Poland.
  • Borgwardt L; Centre for Inherited Metabolic Diseases, Department of Paediatrics and Department of Adolescent Medicine, Copenhagen University Hospital, Rigshospitalet, Copenhagen, Denmark.
  • Gil-Campos M; Center for Genomic Medicine, Copenhagen University Hospital, Rigshospitalet, Copenhagen, Denmark.
  • Guffon N; Metabolism and Pediatric Research Unit, Reina Sofia University Hospital, IMIBIC, University of Cordoba, Unidad de Metabolismo e Investigación Pediátrica, Hospital Universitario Reina Sofía, Universidad de Córdoba, CIBERObn, Córdoba, Spain.
  • Amraoui Y; Reference Center for Inherited Metabolic Diseases, Femme Mere Enfant Hospital, Lyon, France.
  • Geraci S; SphinCS GmbH, Clinical Science for LSD, Hochheim, Germany.
  • Ardigò D; Chiesi Farmaceutici S.p.A, Parma, Italy.
  • Cattaneo F; Chiesi Farmaceutici S.p.A, Parma, Italy.
  • Lund AM; Chiesi Farmaceutici S.p.A, Parma, Italy.
Mol Genet Metab Rep ; 23: 100586, 2020 Jun.
Article en En | MEDLINE | ID: mdl-32292699

Texto completo: 1 Colección: 01-internacional Banco de datos: MEDLINE Idioma: En Revista: Mol Genet Metab Rep Año: 2020 Tipo del documento: Article País de afiliación: Estados Unidos

Texto completo: 1 Colección: 01-internacional Banco de datos: MEDLINE Idioma: En Revista: Mol Genet Metab Rep Año: 2020 Tipo del documento: Article País de afiliación: Estados Unidos