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Natural history, outcome measures and trial readiness in LAMA2-related muscular dystrophy and SELENON-related myopathy in children and adults: protocol of the LAST STRONG study.
Bouman, Karlijn; Groothuis, Jan T; Doorduin, Jonne; van Alfen, Nens; Udink Ten Cate, Floris E A; van den Heuvel, Frederik M A; Nijveldt, Robin; van Tilburg, Willem C M; Buckens, Stan C F M; Dittrich, Anne T M; Draaisma, Jos M T; Janssen, Mirian C H; Kamsteeg, Erik-Jan; van Kleef, Esmee S B; Koene, Saskia; Smeitink, Jan A M; Küsters, Benno; van Tienen, Florence H J; Smeets, Hubert J M; van Engelen, Baziel G M; Erasmus, Corrie E; Voermans, Nicol C.
Afiliação
  • Bouman K; Department of Neurology, Donders Institute for Brain, Cognition and Behaviour, Radboud university medical center, Nijmegen, The Netherlands. Karlijn.bouman@radboudumc.nl.
  • Groothuis JT; Department of Pediatric Neurology, Donders Institute for Brain, Cognition and Behaviour, Amalia Children's Hospital, Radboud university medical center, Nijmegen, The Netherlands. Karlijn.bouman@radboudumc.nl.
  • Doorduin J; Department of Rehabilitation, Donders Institute for Brain, Cognition and Behaviour, Radboud university medical center, Nijmegen, The Netherlands.
  • van Alfen N; Department of Neurology, Donders Institute for Brain, Cognition and Behaviour, Radboud university medical center, Nijmegen, The Netherlands.
  • Udink Ten Cate FEA; Department of Neurology, Donders Institute for Brain, Cognition and Behaviour, Radboud university medical center, Nijmegen, The Netherlands.
  • van den Heuvel FMA; Department of Pediatric cardiology, Amalia Children's Hospital, Radboud university medical center, Nijmegen, The Netherlands.
  • Nijveldt R; Department of Cardiology, Radboud university medical center, Nijmegen, The Netherlands.
  • van Tilburg WCM; Department of Cardiology, Radboud university medical center, Nijmegen, The Netherlands.
  • Buckens SCFM; Department of Radiology, Radboud university medical center, Nijmegen, The Netherlands.
  • Dittrich ATM; Department of Radiology, Radboud university medical center, Nijmegen, The Netherlands.
  • Draaisma JMT; Department of Pediatrics, Amalia Children's Hospital, Radboud university medical center, Nijmegen, The Netherlands.
  • Janssen MCH; Department of Pediatrics, Amalia Children's Hospital, Radboud university medical center, Nijmegen, The Netherlands.
  • Kamsteeg EJ; Department of Internal Medicine, Radboud university medical center, Nijmegen, The Netherlands.
  • van Kleef ESB; Department of Human Genetics, Radboud university medical center, Nijmegen, The Netherlands.
  • Koene S; Department of Neurology, Donders Institute for Brain, Cognition and Behaviour, Radboud university medical center, Nijmegen, The Netherlands.
  • Smeitink JAM; Department of Clinical Genetics, Leiden University Medical Center, Leiden, The Netherlands.
  • Küsters B; Khondrion BV, Nijmegen, The Netherlands.
  • van Tienen FHJ; Department of Pathology, Radboud university medical center, Nijmegen, The Netherlands.
  • Smeets HJM; Department of Toxicogenomics, Maastricht University, Maastricht, The Netherlands.
  • van Engelen BGM; Department of Toxicogenomics, Maastricht University, Maastricht, The Netherlands.
  • Erasmus CE; School for Mental Health and Neurosciences (MHeNS), Maastricht University, Maastricht, the Netherlands.
  • Voermans NC; School for Developmental Biology and Oncology (GROW), Maastricht University, Maastricht, The Netherlands.
BMC Neurol ; 21(1): 313, 2021 Aug 12.
Article em En | MEDLINE | ID: mdl-34384384
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Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Assunto principal: Distrofias Musculares Tipo de estudo: Clinical_trials / Observational_studies Aspecto: Ethics Limite: Adult / Child / Humans Idioma: En Revista: BMC Neurol Ano de publicação: 2021 Tipo de documento: Article
Texto completo
Imprimir
XML
PubMed Links
Buscar no Google

Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Assunto principal: Distrofias Musculares Tipo de estudo: Clinical_trials / Observational_studies Aspecto: Ethics Limite: Adult / Child / Humans Idioma: En Revista: BMC Neurol Ano de publicação: 2021 Tipo de documento: Article