Consensus-based care recommendations for adults with myotonic dystrophy type 1
Open Access
- 1 December 2018
- journal article
- review article
- Published by Ovid Technologies (Wolters Kluwer Health) in Neurology Clinical Practice
- Vol. 8 (6), 507-520
- https://doi.org/10.1212/cpj.0000000000000531
Abstract
Purpose of review Myotonic dystrophy type 1 (DM1) is a severe, progressive genetic disease that affects between 1 in 3,000 and 8,000 individuals globally. No evidence-based guideline exists to inform the care of these patients, and most do not have access to multidisciplinary care centers staffed by experienced professionals, creating a clinical care deficit. Recent findings The Myotonic Dystrophy Foundation (MDF) recruited 66 international clinicians experienced in DM1 patient care to develop consensus-based care recommendations. MDF created a 2-step methodology for the project using elements of the Single Text Procedure and the Nominal Group Technique. The process generated a 4-page Quick Reference Guide and a comprehensive, 55-page document that provides clinical care recommendations for 19 discrete body systems and/or care considerations. Summary The resulting recommendations are intended to help standardize and elevate care for this patient population and reduce variability in clinical trial and study environments.Keywords
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- Myotonic DystrophyNeurologic Clinics, 2014
- Methods of Formal Consensus in Classification/Diagnostic Criteria and Guideline DevelopmentSeminars in Arthritis and Rheumatism, 2011