Consensus-based care recommendations for adults with myotonic dystrophy type 1

Tetsuo Ashizawa; Cynthia Gagnon; William J Groh; Laurie Gutmann; Nicholas E Johnson; Giovanni Meola; Richard Moxley; Shree Pandya; Mark T Rogers; Ericka Simpson; Nathalie Angeard; Guillaume Bassez; Kiera N Berggren; Deepak Bhakta; Marco Bozzali; Ann Broderick; Janice L.B Byrne; Craig Campbell; Edith Cup; John W Day; Elisa De Mattia; Denis Duboc; Tina Duong; Katy Eichinger; Anne-Berit Ekstrom; Baziel van Engelen; Belen Esparis; Bruno Eymard; Marla Ferschl; Shahinaz M Gadalla; Benjamin Gallais; Todd Goodglick; Chad Heatwole; James Hilbert; Venessa Holland; Marie Kierkegaard; Wilma J Koopman; Kari Lane; Daphne Maas; Ami Mankodi; Katherine D Mathews; Darren G Monckton; David Moser; Saman Nazarian; Linda Nguyen; Peg Nopoulos; Richard Petty; Janel Phetteplace; Jack Puymirat; Subha Raman; Louis Richer; Elisabetta Roma; Jacinda Sampson; Valeria Sansone; Benedikt Schoser; Laurie Sterling; Jeffrey Statland; S.H Subramony; Cuixia Tian; Careniña Trujillo; Gordon Tomaselli; Chris Turner; Shannon Venance; Aparajitha Verma; Molly White; Stefan Winblad

doi:10.1212/CPJ.0000000000000531

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Consensus-based care recommendations for adults with myotonic dystrophy type 1

Journal article

Open access

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Consensus-based care recommendations for adults with myotonic dystrophy type 1

Tetsuo Ashizawa, Cynthia Gagnon, William J Groh, Laurie Gutmann, Nicholas E Johnson, Giovanni Meola, Richard Moxley, Shree Pandya, Mark T Rogers, Ericka Simpson, …

Neurology. Clinical practice, Vol.8(6), pp.507-520

12/2018

DOI: 10.1212/CPJ.0000000000000531

PMCID: PMC6294540

PMID: 30588381

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Abstract

Purpose of reviewMyotonic 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 findingsThe 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.SummaryThe resulting recommendations are intended to help standardize and elevate care for this patient population and reduce variability in clinical trial and study environments.

Details

Title: Subtitle: Consensus-based care recommendations for adults with myotonic dystrophy type 1
Creators: Tetsuo Ashizawa
Cynthia Gagnon
William J Groh
Laurie Gutmann
Nicholas E Johnson
Giovanni Meola
Richard Moxley
Shree Pandya
Mark T Rogers
Ericka Simpson
Nathalie Angeard
Guillaume Bassez
Kiera N Berggren
Deepak Bhakta
Marco Bozzali
Ann Broderick
Janice L.B Byrne
Craig Campbell
Edith Cup
John W Day
Elisa De Mattia
Denis Duboc
Tina Duong
Katy Eichinger
Anne-Berit Ekstrom
Baziel van Engelen
Belen Esparis
Bruno Eymard
Marla Ferschl
Shahinaz M Gadalla
Benjamin Gallais
Todd Goodglick
Chad Heatwole
James Hilbert
Venessa Holland
Marie Kierkegaard
Wilma J Koopman
Kari Lane
Daphne Maas
Ami Mankodi
Katherine D Mathews
Darren G Monckton
David Moser
Saman Nazarian
Linda Nguyen
Peg Nopoulos
Richard Petty
Janel Phetteplace
Jack Puymirat
Subha Raman
Louis Richer
Elisabetta Roma
Jacinda Sampson
Valeria Sansone
Benedikt Schoser
Laurie Sterling
Jeffrey Statland
S.H Subramony
Cuixia Tian
Careniña Trujillo
Gordon Tomaselli
Chris Turner
Shannon Venance
Aparajitha Verma
Molly White
Stefan Winblad
Resource Type: Journal article
Publication Details: Neurology. Clinical practice, Vol.8(6), pp.507-520
DOI: 10.1212/CPJ.0000000000000531
PMID: 30588381
PMCID: PMC6294540
ISSN: 2163-0402
eISSN: 2163-0933
Language: English
Date published: 12/2018
Academic Unit: Neurology; Psychiatry; Stead Family Department of Pediatrics; Iowa Neuroscience Institute; Neurology (Pediatrics); General Internal Medicine; Medicine Administration; Internal Medicine
Record Identifier: 9984070815202771

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