Consensus-based care recommendations for adults with myotonic dystrophy type 2. Issue 4 (August 2019)

• Record Type: Journal Article
• Authors: Schoser, Benedikt; Montagnese, Federica; Bassez, Guillaume; Fossati, Barbara; Gamez, Josep; Heatwole, Chad; Hilbert, James; Kornblum, Cornelia; Kostera-Pruszczyk, Anne; Krahe, Ralf; Lusakowska, Anna; Meola, Giovanni; Moxley, Richard; Thornton, Charles; Udd, Bjarne; Formaker, Paul
• Is Part Of: Neurology. Volume 9:Issue 4(2019)
• Publication Date: 2019-08
• Subjects: Nervous system -- Diseases -- Periodicals; Neurology -- Periodicals; Neurology -- United States -- Periodicals; 616.8
• DOI: 10.1212/CPJ.0000000000000645
• Languages: English
• ISSNs: 2163-0402

Abstract

Abstract : 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.