01 Aug 2019
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Consensus-based care recommendations for adults with myotonic dystrophy type 2
Authors:
Benedikt Schoser, MD, Federica Montagnese, MD, Guillaume Bassez, MD, PhD, Barbara Fossati, MD,Josep Gamez, MD, PhD, Chad Heatwole, MD, James Hilbert, MS, Cornelia Kornblum, MD,Anne Kostera-Pruszczyk, MD, PhD, Ralf Krahe, PhD, Anna Lusakowska, MD, PhD, Giovanni Meola, MD, Richard Moxley III, MD, Charles Thornton, MD, Bjarne Udd, MD, PhD, and Paul Formaker, on behalf of the
Myotonic Dystrophy Foundation
Abstract
Purpose of review
Myotonic dystrophy type 2 (DM2) is a rare, progressive multi-
system disease particularly affecting the skeletal muscle. A causal
therapy is not yet available; however, prompt, appropriate symp-
tomatic treatments are essential to limit disease-related complica-
tions. Evidence-based guidelines to assist medical practitioners in
the care of DM2 patients do not exist.
Recent findings
The 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.
Summary
The resulting recommendations will help standardize and improve care for DM2 patients and facilitate appropriate management in centers without neuromuscular specialists.