P1.12 Duchenne muscular dystrophy and optoelectronic plethysmography: A longitudinal study of respiratory function

2011 ◽  
Vol 21 (9-10) ◽  
pp. 645
Author(s):  
M.G. D’Angelo ◽  
M. Romei ◽  
A. Lo Mauro ◽  
E. Marchi ◽  
S. Gandossini ◽  
...  
Keyword(s):  
Longitudinal Study ◽  
Duchenne Muscular Dystrophy ◽  
Muscular Dystrophy ◽  
Respiratory Function ◽  
Optoelectronic Plethysmography

A Longitudinal Study of Quantitative Muscle Strength and Functional Motor Ability in Ambulatory Boys with Duchenne Muscular Dystrophy

2021 ◽  
pp. 1-14
Author(s):  
Cathleen E. Buckon ◽  
Susan E. Sienko ◽  
Eileen G. Fowler ◽  
Anita M. Bagley ◽  
Loretta A. Staudt ◽  
...  
Keyword(s):  
Longitudinal Study ◽  
Duchenne Muscular Dystrophy ◽  
Muscular Dystrophy ◽  
Muscle Strength ◽  
Genetic Disorder ◽  
Knee Extensor ◽  
Standard Of Care ◽  
Rate Of Change ◽  
Isokinetic Dynamometry ◽  
Gross Motor

Background: Duchenne muscular dystrophy (DMD) is an X-linked recessive genetic disorder, that is characterized by progressive muscle degeneration and loss of ambulation between 7–13 years of age. Novel pharmacological agents targeting the genetic defects and disease mechanisms are becoming available; however, corticosteroid (CS) therapy remains the standard of care. Objective: The purpose of this longitudinal study was to elucidate the effect of CS therapy on the rate of muscle strength and gross motor skill decline in boys with DMD and assess the sensitivity of selected outcome measures. Methods: Eighty-four ambulatory boys with DMD (49–180 months), 70 on CS, 14 corticosteroid naïve (NCS), participated in this 8-year multi-site study. Outcomes included; isokinetic dynamometry, the Standing (STD) and Walking/Running/jumping (WRJ) dimensions of the Gross Motor Function Measure (GMFM), and Timed Function Tests (TFTs). Nonlinear mixed modeling procedures determined the rate of change with age and the influence of steroids. Results: Despite CS therapy the rate of decline in strength with age was significant in all muscle groups assessed. CS therapy significantly slowed decline in knee extensor strength, as the NCS group declined at 3x the rate of the CS group. Concurrently, WRJ skills declined in the NCS group at twice the rate of the CS group. 4-stair climb and 10 meter walk/run performance was superior in the boys on CS therapy. Conclusion: CS therapy slowed the rate of muscle strength decline and afforded longer retention of select gross motor skills in boys on CS compared to boys who were NCS. Isokinetic dynamometry, Walk/Run/Jump skills, and select TFTs may prove informative in assessing the efficacy of new therapeutics in ambulatory boys with DMD.

scholarly journals Genetic modifiers of respiratory function in Duchenne muscular dystrophy

2020 ◽  
Vol 7 (5) ◽  
pp. 786-798
Author(s):  
Luca Bello ◽  
Grazia D’Angelo ◽  
Matteo Villa ◽  
Aurora Fusto ◽  
Sara Vianello ◽  
...  
Keyword(s):  
Duchenne Muscular Dystrophy ◽  
Muscular Dystrophy ◽  
Respiratory Function ◽  
Genetic Modifiers

scholarly journals Early Neurodevelopmental Findings Predict School Age Cognitive Abilities in Duchenne Muscular Dystrophy: A Longitudinal Study

PLoS ONE ◽  
2015 ◽  
Vol 10 (8) ◽  
pp. e0133214 ◽  
Author(s):  
Daniela Chieffo ◽  
Claudia Brogna ◽  
Angela Berardinelli ◽  
Grazia D’Angelo ◽  
Maria Mallardi ◽  
...  
Keyword(s):  
Longitudinal Study ◽  
Duchenne Muscular Dystrophy ◽  
Muscular Dystrophy ◽  
Cognitive Abilities ◽  
School Age

Respiratory function in eteplirsen-treated Duchenne muscular dystrophy (DMD) patients compared to natural history

2016 ◽  
Vol 26 ◽  
pp. S154
Author(s):  
B. Kinane ◽  
O. Mayer ◽  
L. Lowes ◽  
N. Khan ◽  
K. Gallitano ◽  
...  
Keyword(s):  
Duchenne Muscular Dystrophy ◽  
Muscular Dystrophy ◽  
Natural History ◽  
Respiratory Function

EP.80The effects of steroid treatment on respiratory function in adults with Duchenne muscular dystrophy after loss of ambulation

2019 ◽  
Vol 29 ◽  
pp. S176
Author(s):  
A. Pietrusz ◽  
M. Desikan ◽  
F. Trucco ◽  
A. Manzur ◽  
F. Muntoni ◽  
...  
Keyword(s):  
Duchenne Muscular Dystrophy ◽  
Muscular Dystrophy ◽  
Respiratory Function ◽  
Steroid Treatment
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