[en] There has been tremendous progress in treatment of neuromuscular diseases over the last 20 years, which has transformed the natural history of these severely debilitating conditions. Although the factors that determine the response to therapy are many and in some instance remain to be fully elucidated, early treatment clearly has a major impact on patient outcomes across a number of inherited neuromuscular conditions. To improve patient care and outcomes, clinicians should be aware of neuromuscular conditions that require prompt treatment initiation. This review describes data that underscore the importance of early treatment of children with inherited neuromuscular conditions with an emphasis on data resulting from newborn screening efforts.
Disciplines :
Pediatrics
Author, co-author :
Mackels, Laurane; MDUK Oxford Neuromuscular Centre, Department of Paediatrics, University of Oxford, Oxford, United Kingdom ; Adult Neurology Department, Citadelle Hospital, Liège, Belgium
Servais, Laurent ; Université de Liège - ULiège > Département des sciences cliniques ; MDUK Oxford Neuromuscular Centre, Department of Paediatrics, University of Oxford & NIHR Oxford Biomedical Research Centre, Oxford, United Kingdom
Language :
English
Title :
The Importance of Early Treatment of Inherited Neuromuscular Conditions.
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