Consensus-based care recommendations for adults with myotonic dystrophy type 2

Benedikt Schoser, Federica Montagnese, Guillaume Bassez, Barbara Fossati, Josep Gamez, Chad Heatwole, James Hilbert, Cornelia Kornblum, Anne Kostera-Pruszczyk, Ralf Krahe, Anna Lusakowska, Giovanni Meola, Richard Moxley, Charles Thornton, Bjarne Udd, Paul Formaker

Research output: Contribution to journalReview articlepeer-review

33 Scopus citations

Abstract

Purpose of reviewMyotonic dystrophy type 2 (DM2) is a rare, progressive multisystem disease particularly affecting the skeletal muscle. A causal therapy is not yet available; however, prompt, appropriate symptomatic treatments are essential to limit disease-related complications. Evidence-based guidelines to assist medical practitioners in the care of DM2 patients do not exist.Recent findingsThe Myotonic Dystrophy Foundation (MDF) previously worked with an international group of 66 clinicians to develop consensus-based care recommendations for myotonic dystrophy type 1. Following a similar approach, the MDF recruited 15 international clinicians with long-standing experience in the care of DM2 patients to develop consensus-based care recommendations. The single text procedure was adopted. This process generated a 4-page Quick Reference Guide and a comprehensive 55-page document that provides care recommendations for DM2 patients.SummaryThe resulting recommendations will help standardize and improve care for DM2 patients and facilitate appropriate management in centers without neuromuscular specialists.

Original languageEnglish (US)
Pages (from-to)343-353
Number of pages11
JournalNeurology: Clinical Practice
Volume9
Issue number4
DOIs
StatePublished - Aug 1 2019
Externally publishedYes

ASJC Scopus subject areas

  • Clinical Neurology

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