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Functional assessment tools in children with Pompe disease: A pilot comparative study to identify suitable outcome measures for the standard of care.
Ricci, Federica; Brusa, Chiara; Rossi, Francesca; Rolle, Enrica; Placentino, Valeria; Berardinelli, Angela; Pagliardini, Veronica; Porta, Francesco; Spada, Marco; Mongini, Tiziana.
Afiliação
  • Ricci F; Department of Pediatrics, Division of Child Neurology and Psychiatry, Turin University Hospital, Piazza Polonia 94, 10126, Turin, Italy. Electronic address: federica.ricci@unito.it.
  • Brusa C; Department of Pediatrics, Division of Child Neurology and Psychiatry, Turin University Hospital, Piazza Polonia 94, 10126, Turin, Italy.
  • Rossi F; Department of Pediatrics, Division of Child Neurology and Psychiatry, Turin University Hospital, Piazza Polonia 94, 10126, Turin, Italy.
  • Rolle E; Department of Pediatrics, Division of Child Neurology and Psychiatry, Turin University Hospital, Piazza Polonia 94, 10126, Turin, Italy.
  • Placentino V; Department of Pediatrics, Division of Child Neurology and Psychiatry, Turin University Hospital, Piazza Polonia 94, 10126, Turin, Italy.
  • Berardinelli A; Division of Childhood and Adolescence Neurology, IRCCS Mondino, via Mondino 2, 27100, Pavia, Italy.
  • Pagliardini V; Department of Pediatrics, Division of Metabolic Diseases, Turin University Hospital, Piazza Polonia 94, 10126, Turin, Italy.
  • Porta F; Department of Pediatrics, Division of Metabolic Diseases, Turin University Hospital, Piazza Polonia 94, 10126, Turin, Italy.
  • Spada M; Department of Pediatrics, Division of Metabolic Diseases, Turin University Hospital, Piazza Polonia 94, 10126, Turin, Italy.
  • Mongini T; Department of Neuroscience, Division of Neurology and Neuromuscular Diseases, Turin University Hospital, Corso Bramante 88/90, 10126, Turin, Italy.
Eur J Paediatr Neurol ; 22(6): 1103-1109, 2018 Nov.
Article em En | MEDLINE | ID: mdl-30166092

Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Assunto principal: Doença de Depósito de Glicogênio Tipo II / Avaliação de Resultados em Cuidados de Saúde / Progressão da Doença Tipo de estudo: Guideline / Prognostic_studies Aspecto: Patient_preference Limite: Child / Child, preschool / Female / Humans / Infant / Male Idioma: En Revista: Eur J Paediatr Neurol Assunto da revista: NEUROLOGIA / PEDIATRIA Ano de publicação: 2018 Tipo de documento: Article País de publicação: Reino Unido

Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Assunto principal: Doença de Depósito de Glicogênio Tipo II / Avaliação de Resultados em Cuidados de Saúde / Progressão da Doença Tipo de estudo: Guideline / Prognostic_studies Aspecto: Patient_preference Limite: Child / Child, preschool / Female / Humans / Infant / Male Idioma: En Revista: Eur J Paediatr Neurol Assunto da revista: NEUROLOGIA / PEDIATRIA Ano de publicação: 2018 Tipo de documento: Article País de publicação: Reino Unido