1. European Cross-Sectional Survey of Current Care Practices for Duchenne Muscular Dystrophy Reveals Regional and Age-Dependent Differences
- Author
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Maria de los Angeles Beytía, Anna Lusakowska, Petr Vondráček, Birgit F. Steffensen, Sam Doerken, K. Gramsch, Agnes Herczegfalvi, Hanns Lochmüller, Kate Bushby, Veronika Karcagi, Anna Kostera-Pruszczyk, Marta Garami, Adrian Tassoni, Teodora Chamova, Lenka Mrázová, Lenka Pavlovska, Sunil Rodger, Velina Guergueltcheva, Rachel Thompson, J. Vry, Jes Rahbek, Janbernd Kirschner, Jana Strenková, Ivailo Tournev, and Anna Kamińska
- Subjects
Male ,Research Report ,0301 basic medicine ,Gerontology ,standards of care ,Cross-sectional study ,Duchenne muscular dystrophy ,Alternative medicine ,Age dependent ,functional status ,0302 clinical medicine ,Adrenal Cortex Hormones ,Surveys and Questionnaires ,Milestone (project management) ,Medicine ,Registries ,Practice Patterns, Physicians' ,Young adult ,Child ,Age Factors ,Standard of Care ,Middle Aged ,Respiratory Function Tests ,3. Good health ,Patient management ,Europe ,Neurology ,Echocardiography ,Child, Preschool ,Practice Guidelines as Topic ,Guideline Adherence ,Psychosocial ,Adult ,musculoskeletal diseases ,congenital, hereditary, and neonatal diseases and abnormalities ,medicine.medical_specialty ,Adolescent ,Young Adult ,03 medical and health sciences ,Humans ,Physical Therapy Modalities ,business.industry ,Infant ,medicine.disease ,nervous system diseases ,Muscular Dystrophy, Duchenne ,Cross-Sectional Studies ,030104 developmental biology ,Physical therapy ,Neurology (clinical) ,business ,corticosteroid treatment ,030217 neurology & neurosurgery - Abstract
Background: Publication of comprehensive clinical care guidelines for Duchenne muscular dystrophy (DMD) in 2010 was a milestone for DMD patient management. Our CARE-NMD survey investigates the neuromuscular, medical, and psychosocial care of DMD patients in Europe, and compares it to the guidelines. Methods: A cross-sectional survey of 1677 patients contacted via the TREAT-NMD patient registries was conducted using self-report questionnaires in seven European countries. Results: Survey respondents were 861 children and 201 adults. Data describe a European DMD population with mean age of 13.0 years (range 0.8–46.2) of whom 53% had lost ambulation (at 10.3 years of age, median). Corticosteroid medication raised the median age for ambulatory loss from 10.1 years in patients never medicated to 11.4 years in patients who received steroids (p
- Published
- 2016