Consensus statement on standard of care for congenital muscular dystrophies

Ching H. Wang, Carsten G. Bonnemann, Anne Rutkowski, Thomas Sejersen, Jonathan Bellini, Vanessa Battista, Julaine M. Florence, Ulrike Schara, Pamela M. Schuler, Karim Wahbi, Annie Aloysius, Robert O. Bash, Christophe Béroud, Enrico Bertini, Kate Bushby, Ronald D. Cohn, Anne M. Connolly, Nicolas Deconinck, Isabelle Desguerre, Michelle EagleBrigitte Estournet-Mathiaud, Ana Ferreiro, Albert Fujak, Nathalie Goemans, Susan T. Iannaccone, Patricia Jouinot, Marion Main, Paola Melacini, Wolfgang Mueller-Felber, Francesco Muntoni, Leslie L. Nelson, Jes Rahbek, Susana Quijano-Roy, Caroline Sewry, Kari Storhaug, Anita Simonds, Brian Tseng, Jiri Vajsar, Andrea Vianello, Reinhard Zeller

Research output: Contribution to journalArticlepeer-review

176 Scopus citations


Congenital muscular dystrophies are a group of rare neuromuscular disorders with a wide spectrum of clinical phenotypes. Recent advances in understanding the molecular pathogenesis of congenital muscular dystrophy have enabled better diagnosis. However, medical care for patients with congenital muscular dystrophy remains very diverse. Advances in many areas of medical technology have not been adopted in clinical practice. The International Standard of Care Committee for Congenital Muscular Dystrophy was established to identify current care issues, review literature for evidence-based practice, and achieve consensus on care recommendations in 7 areas: diagnosis, neurology, pulmonology, orthopedics/rehabilitation, gastroenterology/ nutrition/speech/oral care, cardiology, and palliative care. To achieve consensus on the care recommendations, 2 separate online surveys were conducted to poll opinions from experts in the field and from congenital muscular dystrophy families. The final consensus was achieved in a 3-day workshop conducted in Brussels, Belgium, in November 2009. This consensus statement describes the care recommendations from this committee.

Original languageEnglish (US)
Pages (from-to)1559-1581
Number of pages23
JournalJournal of child neurology
Issue number12
StatePublished - Dec 2010


  • congenital muscular dystrophy
  • standard of care

ASJC Scopus subject areas

  • Pediatrics, Perinatology, and Child Health
  • Clinical Neurology


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