RT Journal Article
SR Electronic
T1 Consensus-based care recommendations for adults with myotonic dystrophy type 2
JF Neurology: Clinical Practice
FD Lippincott Williams & Wilkins
SP 343
OP 353
DO 10.1212/CPJ.0000000000000645
VO 9
IS 4
A1 Schoser, Benedikt
A1 Montagnese, Federica
A1 Bassez, Guillaume
A1 Fossati, Barbara
A1 Gamez, Josep
A1 Heatwole, Chad
A1 Hilbert, James
A1 Kornblum, Cornelia
A1 Kostera-Pruszczyk, Anne
A1 Krahe, Ralf
A1 Lusakowska, Anna
A1 Meola, Giovanni
A1 Moxley, Richard
A1 Thornton, Charles
A1 Udd, Bjarne
A1 Formaker, Paul
A1 on behalf of the Myotonic Dystrophy Foundation
YR 2019
UL http://cp.neurology.org/content/9/4/343.abstract
AB Purpose of review Myotonic 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 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.