MD Tetsuo Ashizawa, PhD Cynthia Gagnon, M. M. William J. Groh, MD Laurie Gutmann, MD Nicholas E. Johnson, MD Giovanni Meola, MD Richard Moxley III, Dpt Shree Pandya, MD Mark T. Rogers, MD Ericka Simpson, PhD Nathalie Angeard, MD Guillaume Bassez, Kiera N. Berggren PhD, M. Bhakta, M. Md, A. Md, Moemenbellah-Fard Md, C. Md, MD Edith Cup, Jonathan Phd, Elisa De Mattia PhD, D. Pt, T. Md, PhDc Katy Eichinger Mpt, Anne-Berit Ekstrom PhD, Ba Phd, Belen Esparis PhD, MD Bruno Eymard, M. Md, S. M. Md, Benjamin Gallais PhD, PhD Todd Goodglick, C. Md, J. Md, V. Ms, M. Md, Wilma J. Koopman PhD, Kari Lane PhD, D. Rd, M. Pt, M. Mathews, D. G. Md, David Moser PhD, Saman Nazarian PhD, Peter Nguyen, P. Md, R. Md, J. Md, J. Ms, P. Raman, L. Md, Elisabetta Roma PhD, J. Md, P. Sansone, Benedikt Schoser PhD, L. Md, J. Ms, MD S.H.Subramony, CuixiaTian Md, C. Md, MSNGordonTomaselli Rn, ChrisTurner Md, PhD Shannon Venance, Aparajitha Verma PhD, M. Md, MA Stefan Winblad
2018.9.13Neurology-Clinical Practice
tlooto 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.
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.
Citation format
ASHIZAWA, MD Tetsuo, et al. Consensus-based care recommendations for adults with myotonic dystrophy type 1. Neurology-Clinical Practice, 2018, 8: 507–520.