Clinical trial in duchenne dystrophy. I. The design of the protocol

Brooke, Michael H.; Griggs, Robert C.; Mendell, Jerry R.; Fenichel, Gerald M.; Shumate, Jack B.; Pellegrino, Richard J.

doi:10.1002/mus.880040304

Cited by 498 publications

(337 citation statements)

References 6 publications

Supporting

Mentioning

311

Contrasting

Unclassified

Order By: Relevance

“…Functional measures developed specifically for older patients with neuromuscular diseases have included the Vignos lower extremity classification scale [16], the Brooke upper extremity scale [17,18], various timed functional assessments [17], and The Hammersmith motor ability scale [19]. More recently the EK scale [20] was developed for non-ambulatory patients with Duchenne's muscular dystrophy (DMD) and SMA, the Wee Fim was utilized to quantify function in children with SMA in Hong Kong [11] and the functional research scale for ALS (FRS-ALS) scale was utilized in clinical trials with adult patients with ALS [21,22].…”

Section: Introductionmentioning

confidence: 99%

A modified Hammersmith functional motor scale for use in multi-center research on spinal muscular atrophy

Krosschell

Maczulski²,

Crawford

et al. 2006

Neuromuscular Disorders

View full text Add to dashboard Cite

The Hammersmith functional motor scale for children with spinal muscular atrophy was modified to establish a standard measure of functional ability in children with non-ambulant spinal muscular atrophy types 2 and 3 in a longitudinal multi-center clinical trial. This study assessed the intra-and interrater reliability and the test-retest stability of a modified version of the scale. Both intra-and interrater reliability were established. Results indicate that the scale is reliable and stable over a 6 month period. Reliability was maintained when patient sample criteria were expanded to include children younger than 30 months and children with popliteal angles greater than 20°. These data establish the modified Hammersmith functional motor scale for children with spinal muscular atrophy as a reliable instrument for use in multi-center treatment trials in non-ambulant spinal muscular atrophy children. Our data provides additional support for the use of original scale items in terms of ease of administration, usefulness and reliability, while incorporating modifications to optimize its use in a multi-center clinical research setting.

show abstract

Section: Introductionmentioning

confidence: 99%

A modified Hammersmith functional motor scale for use in multi-center research on spinal muscular atrophy

Krosschell

Maczulski²,

Crawford

et al. 2006

Neuromuscular Disorders

View full text Add to dashboard Cite

show abstract

“…Studies have shown the relevance of motor function follow up to monitor DMD accurately 2,3,4 . Global motor scales should be complemented by functional tasks assessment because, despite the continued decline in muscle strength, children with DMD continue to perform the activities using compensatory movements 5,6 .…”

mentioning

confidence: 99%

How often should sitting and rising from a chair be evaluated in patients with Duchenne muscular dystrophy?

Hukuda

Caromano

Escórcio

et al. 2017

Arq. Neuro-Psiquiatr.

View full text Add to dashboard Cite

Objective: To determine how often sitting/rising from a chair should be assessed in Duchenne muscular dystrophy (DMD) patients to avoid redundant/missing data. Methods: Sitting/rising from a chair was evaluated in 26 DMD children (5-12 yrs), in three-month intervals, over twelve months, with the Functional Evaluation Scale (domain sitting/rising from a chair). Scores were compared by effect sizes (ES) and standardized response means (SRM) (responsiveness analysis). Results: Sit-to-stand showed low-to-moderate responsiveness in three-month intervals

show abstract

“…(i) Semi-quantitative measurements (manual testing, timed scores during motor acts) are performed in routine clinical practice and afford a functional evaluation (Edwards & Hide, 1977;Brooke et al 1981;Aitkens et al 1989). For instance, manual testing (scored from 0 to 5) is based on an international graduated scale of muscle ability: MRC scale (Medical Research Council, 1943) reorganized from 0 to 10 (Brooke et al 1981).…”

mentioning

confidence: 99%

Muscle and joint elastic properties during elbow flexion in Duchenne muscular dystrophy

Cornu

Goubel

Fardeau

2001

The Journal of Physiology

View full text Add to dashboard Cite

Duchenne muscular dystrophy (DMD) is characterized by a direct effect on muscle fibres leading to muscle atrophy and weakness due to a lack of muscle dystrophin. Dystrophin is a sarcolemma cytoskeletal protein whose deficit leads to segmental necrosis of the muscle fibre. DMD is thus characterized by a decrease in the number of muscle fibres, which are progressively replaced by fibroadipose tissue. The first observed clinical signs relate to difficulties in running or climbing stairs and calf hypertrophy. Gait then becomes lordotic and waddling. Between 6 and 11 years of age, limb and torso muscles progressively and steadily decrease in strength. Loss of ambulation occurs (average 10.5 years of age) and all torso and limb muscles decrease in size.The assessment of muscle mechanical properties in DMD patients is mostly limited to a more or less sensitive evaluation of strength production, generally in static conditions and occasionally during a locomotor act. Two kinds of non-invasive technique are generally used to characterize muscle functional properties in DMD patients. (i) Semi-quantitative measurements (manual testing, timed scores during motor acts) are performed in routine clinical practice and afford a functional evaluation (Edwards & Hide, 1977;Brooke et al. 1981;Aitkens et al. 1989). For instance, manual testing (scored from 0 to 5) is based on an international graduated scale of muscle ability: MRC scale (Medical Research Council, 1943) reorganized from 0 to 10 (Brooke et al. 1981).(ii) Quantitative measurements (manual dynamometry) can be performed in some muscle groups and give a maximal voluntary contraction (MVC) measurement (Munsat, 1989).Understanding of the mechanisms responsible for muscle adaptation to altered functional demand (pathology or training) requires quantification of muscle mechanical properties. For instance, muscle contractility and muscle elasticity were experimentally found to be sensitive to periods of hyperactivity as well as hypoactivity (Goubel & Marini, 1987; Almeida Silveira et al. 1994;Canon & Goubel, 1995 1. Maximal voluntary contraction (MVC), series elastic stiffness and total joint stiffness during elbow flexion were investigated in healthy boys and in boys with Duchenne muscular dystrophy (DMD) in order to assess changes in mechanical properties induced by the disease.2. Two methods were used to perform stiffness measurements: (i) the application of sinusoidal perturbations to the joint during flexion efforts, allowing the calculation of total joint stiffness; (ii) the use of quick-release movements of the elbow, which had previously been maintained in isometric contraction, allowing the calculation of series elastic stiffness. In each case, stiffness was linearly related to torque, leading to the calculation of a normalized stiffness index as the slope of this stiffness-torque relationship.3. As expected, mean MVC was found to be much higher for healthy boys (20.02 ± 5.20 N m) than for DMD patients (3.09 ± 2.44 N m). Furthermore, the results showed that it was poss...

show abstract

Clinical trial in duchenne dystrophy. I. The design of the protocol

Cited by 498 publications

References 6 publications

A modified Hammersmith functional motor scale for use in multi-center research on spinal muscular atrophy

A modified Hammersmith functional motor scale for use in multi-center research on spinal muscular atrophy

How often should sitting and rising from a chair be evaluated in patients with Duchenne muscular dystrophy?

Muscle and joint elastic properties during elbow flexion in Duchenne muscular dystrophy

Contact Info

Product

Resources

About