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.

Autor: Bouman K; Department of Neurology, Donders Institute for Brain, Cognition and Behaviour, Radboud university medical center, Nijmegen, The Netherlands. Karlijn.bouman@radboudumc.nl.; 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., Groothuis JT; Department of Rehabilitation, Donders Institute for Brain, Cognition and Behaviour, Radboud university medical center, Nijmegen, The Netherlands., Doorduin J; Department of Neurology, 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 Pediatric cardiology, Amalia Children's Hospital, Radboud university medical center, Nijmegen, The Netherlands., van den Heuvel FMA; Department of Cardiology, Radboud university medical center, Nijmegen, The Netherlands., Nijveldt R; Department of Cardiology, Radboud university medical center, Nijmegen, The Netherlands., van Tilburg WCM; Department of Radiology, Radboud university medical center, Nijmegen, The Netherlands., Buckens SCFM; Department of Radiology, Radboud university medical center, Nijmegen, The Netherlands., Dittrich ATM; Department of Pediatrics, Amalia Children's Hospital, 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 Internal Medicine, Radboud university medical center, Nijmegen, The Netherlands., Kamsteeg EJ; Department of Human Genetics, Radboud university medical center, Nijmegen, The Netherlands., van Kleef ESB; Department of Neurology, Donders Institute for Brain, Cognition and Behaviour, Radboud university medical center, Nijmegen, The Netherlands., Koene S; Department of Clinical Genetics, Leiden University Medical Center, Leiden, The Netherlands., Smeitink JAM; Khondrion BV, Nijmegen, The Netherlands., Küsters B; Department of Pathology, Radboud university medical center, Nijmegen, The Netherlands., van Tienen FHJ; Department of Toxicogenomics, Maastricht University, Maastricht, The Netherlands., Smeets HJM; Department of Toxicogenomics, Maastricht University, Maastricht, The Netherlands.; School for Mental Health and Neurosciences (MHeNS), Maastricht University, Maastricht, the Netherlands.; School for Developmental Biology and Oncology (GROW), Maastricht University, Maastricht, The Netherlands., van Engelen BGM; Department of Neurology, Donders Institute for Brain, Cognition and Behaviour, Radboud university medical center, Nijmegen, The Netherlands., Erasmus CE; Department of Pediatric Neurology, Donders Institute for Brain, Cognition and Behaviour, Amalia Children's Hospital, Radboud university medical center, Nijmegen, The Netherlands., Voermans NC; Department of Neurology, Donders Institute for Brain, Cognition and Behaviour, Radboud university medical center, Nijmegen, The Netherlands.

Publikováno v: BMC neurology [BMC Neurol] 2021 Aug 12; Vol. 21 (1), pp. 313. Date of Electronic Publication: 2021 Aug 12.