Getting ready for clinical trials in myotonic dystrophy type 1 with the validation of functional outcome measures

2017 ◽  
Vol 27 ◽  
pp. S180
Author(s):  
A. Jimenez-Moreno ◽  
N. Nikolenko ◽  
D. Moat ◽  
A. Mayhew ◽  
C. Massey ◽  
...  
Keyword(s):  
Clinical Trials ◽  
Functional Outcome ◽  
Myotonic Dystrophy ◽  
Outcome Measures ◽  
Myotonic Dystrophy Type 1 ◽  
Myotonic Dystrophy Type ◽  
Functional Outcome Measures

Report of the first Outcome Measures in Myotonic Dystrophy type 1 (OMMYD-1) international workshop

2013 ◽  
Vol 23 (12) ◽  
pp. 1056-1068 ◽  
Author(s):  
Cynthia Gagnon ◽  
Giovanni Meola ◽  
Luc J. Hébert ◽  
Jack Puymirat ◽  
Luc Laberge ◽  
...  
Keyword(s):  
Myotonic Dystrophy ◽  
Outcome Measures ◽  
International Workshop ◽  
Myotonic Dystrophy Type 1 ◽  
Myotonic Dystrophy Type

scholarly journals Muscle MRI in Myotonic Dystrophy type 1 (DM1): refining muscle involvement and implications for clinical trials

2021 ◽  
Author(s):  
Matteo Garibaldi ◽  
Tommaso Nicoletti ◽  
Elisabetta Bucci ◽  
Laura Fionda ◽  
Luca Leonardi ◽  
...  
Keyword(s):  
Clinical Trials ◽  
Myotonic Dystrophy ◽  
Myotonic Dystrophy Type 1 ◽  
Myotonic Dystrophy Type ◽  
Muscle Mri ◽  
Muscle Involvement

Report of the second Outcome Measures in Myotonic Dystrophy type 1 (OMMYD-2) international workshop San Sebastian, Spain, October 16, 2013

2015 ◽  
Vol 25 (7) ◽  
pp. 603-616 ◽  
Author(s):  
Cynthia Gagnon ◽  
Giovanni Meola ◽  
Luc J. Hébert ◽  
Luc Laberge ◽  
Mario Leone ◽  
...  
Keyword(s):  
Myotonic Dystrophy ◽  
Outcome Measures ◽  
International Workshop ◽  
Myotonic Dystrophy Type 1 ◽  
Myotonic Dystrophy Type ◽  
San Sebastian

Effects and Acceptability of an Individualized Home-Based 10-Week Training Program in Adults with Myotonic Dystrophy Type 1

2021 ◽  
Vol 8 (1) ◽  
pp. 137-149
Author(s):  
Isabelle Lessard ◽  
Sébastien Gaboury ◽  
Cynthia Gagnon ◽  
Kévin Bouchard ◽  
Kévin Chapron ◽  
...  
Keyword(s):  
Training Program ◽  
Myotonic Dystrophy ◽  
Outcome Measures ◽  
Functional Mobility ◽  
Myotonic Dystrophy Type 1 ◽  
Myotonic Dystrophy Type ◽  
Walk Test ◽  
Adult Form ◽  
Home Based

Background: Muscle weakness is a cardinal sign of myotonic dystrophy type 1, causing important functional mobility limitations and increasing the risk of falling. As a non-pharmacological, accessible and safe treatment for this population, strength training is an intervention of choice. Objective: To document the effects and acceptability of an individualized semi-supervised home-based exercise program on functional mobility, balance and lower limb strength, and to determine if an assistive training device has a significant impact on outcomes. Methods: This study used a pre-post test design and men with the adult form of DM1 were randomly assigned to the control or device group. The training program was performed three times a week for 10 weeks and included three exercises (sit-to-stand, squat, and alternated lunges). Outcome measures included maximal isometric muscle strength, 10-Meter Walk Test, Mini-BESTest, 30-Second Chair Stand Test and 6-minute walk test. Results: No outcome measures showed a significant difference, except for the strength of the knee flexors muscle group between the two assessments. All participants improved beyond the standard error of measurement in at least two outcome measures. The program and the device were well accepted and all participants reported many perceived improvements at the end of the program. Conclusions: Our results provide encouraging data on the effects and acceptability of a home-based training program for men with the adult form of DM1. These programs would reduce the financial burden on the health system while improving the clinical services offered to this population.

Sign in / Sign up

Export Citation Format

Share Document