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Patients' view on gene therapy development for lysosomal storage disorders: a qualitative study.
Eskes, Eline C B; Beishuizen, Cathrien R L; Corazolla, Eleonore M; van Middelaar, Tessa; Brands, Marion M M G; Dekker, Hanka; van de Mheen, Erica; Langeveld, Mirjam; Hollak, Carla E M; Sjouke, Barbara.
Afiliação
  • Eskes ECB; Amsterdam UMC, University of Amsterdam, Endocrinology and Metabolism, Meibergdreef 9, Amsterdam, The Netherlands.
  • Beishuizen CRL; Amsterdam Gastroenterology Endocrinology Metabolism, Inborn Errors of Metabolism, Meibergdreef 9, Amsterdam, The Netherlands.
  • Corazolla EM; Amsterdam UMC, University of Amsterdam, General Practice, Meibergdreef 9, Amsterdam, The Netherlands.
  • van Middelaar T; Amsterdam UMC, University of Amsterdam, Endocrinology and Metabolism, Meibergdreef 9, Amsterdam, The Netherlands.
  • Brands MMMG; Amsterdam Gastroenterology Endocrinology Metabolism, Inborn Errors of Metabolism, Meibergdreef 9, Amsterdam, The Netherlands.
  • Dekker H; Amsterdam UMC, University of Amsterdam, Laboratory Genetic Metabolic Diseases, Meibergdreef 9, Amsterdam, The Netherlands.
  • van de Mheen E; Amsterdam UMC, University of Amsterdam, Neurology, Meibergdreef 9, Amsterdam, The Netherlands.
  • Langeveld M; Amsterdam Gastroenterology Endocrinology Metabolism, Inborn Errors of Metabolism, Meibergdreef 9, Amsterdam, The Netherlands.
  • Hollak CEM; Amsterdam UMC, University of Amsterdam, Emma Children's Hospital, Department of Pediatrics, Division of Metabolic Diseases, Meibergdreef 9, Amsterdam, The Netherlands.
  • Sjouke B; VKS, The Dutch Patient Association for Inherited Metabolic Diseases, Zwolle, The Netherlands.
Orphanet J Rare Dis ; 17(1): 383, 2022 10 21.
Article em En | MEDLINE | ID: mdl-36271424

Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Assunto principal: Doenças por Armazenamento dos Lisossomos / Doença de Fabry / Mucopolissacaridose III / Doença de Gaucher Tipo de estudo: Prognostic_studies / Qualitative_research Aspecto: Ethics Limite: Adult / Humans Idioma: En Revista: Orphanet J Rare Dis Assunto da revista: MEDICINA Ano de publicação: 2022 Tipo de documento: Article País de afiliação: Holanda País de publicação: Reino Unido

Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Assunto principal: Doenças por Armazenamento dos Lisossomos / Doença de Fabry / Mucopolissacaridose III / Doença de Gaucher Tipo de estudo: Prognostic_studies / Qualitative_research Aspecto: Ethics Limite: Adult / Humans Idioma: En Revista: Orphanet J Rare Dis Assunto da revista: MEDICINA Ano de publicação: 2022 Tipo de documento: Article País de afiliação: Holanda País de publicação: Reino Unido