Becker??s Muscular Dystrophy

McDonald, Craig; Abresch, Richard T.; Carter, G. T.; Fowler, William M.; Johnson, Ernest H.; Kilmer, David D.

doi:10.1097/00002060-199509001-00004

Cited by 62 publications

(5 citation statements)

References 0 publications

Supporting

Mentioning

Contrasting

Order By: Relevance

“…There was no dilated cardiomyopathy noted in this case, which would be an unusual presentation for BMD or DMD because dilation becomes more apparent with age in both of these. 9,10 Neck muscle involvement, which is common in BMD and DMD 11 is also lacking in this case. Therefore, based on the gross pattern of affected muscles, the histological characteristics with the calculated fat to fascicle percentage, the apparent lack of cardiac involvement, as well as age and size of the patient, a form of adult-onset Limb Girdle Muscular Dystrophy (LGMD) is suspected.…”

Section: Discussionmentioning

confidence: 68%

Dystrophic muscle distribution in late-stage muscular dystrophy

et al. 2020

View full text Add to dashboard Cite

Section: Discussionmentioning

confidence: 68%

Dystrophic muscle distribution in late-stage muscular dystrophy

et al. 2020

View full text Add to dashboard Cite

“…In our study, we had 19 participants with some lifetime corticosteroid exposure (mean[median] lifetime exposure at last visit = 5.6 [ 2.1 ] years). Of these 19, ten participants had exposure to corticosteroids during the study (mean[median] lifetime exposure at last visit = 7.0 [ 3.3 ] years).…”

Section: Resultsmentioning

confidence: 99%

“…The data analysis in this report adds to the limited prior experience with prospectively collected, longi-tudinal data on functional outcomes for patients with BMD [1][2][3][4]11]. Of these studies, Bello et al [11] had sufficiently large groups with either a DMD deletion ending in exon 51 or in exon 45.…”

Section: Discussionmentioning

confidence: 98%

“…Becker muscular dystrophy (BMD) is an allelic disease to Duchenne muscular dystrophy (DMD), and both disorders are caused by mutations in the DMD gene. BMD has a far greater age range at onset and more variability in progressive motor decline compared to DMD [1][2][3][4]. Therefore, the Cooperative International Neuromuscular Research Group (CINRG) conducted a prospective natural history study enrolling 83 participants, both ambulatory and non-ambulatory, with BMD.…”

Section: Introductionmentioning

confidence: 99%

See 1 more Smart Citation

Findings from the Longitudinal CINRG Becker Natural History Study

Clemens,

Gordish-Dressman,

Niizawa

et al. 2024

JND

Self Cite

View full text Add to dashboard Cite

Background: Becker muscular dystrophy is an X-linked, genetic disorder causing progressive degeneration of skeletal and cardiac muscle, with a widely variable phenotype. Objective: A 3-year, longitudinal, prospective dataset contributed by patients with confirmed Becker muscular dystrophy was analyzed to characterize the natural history of this disorder. A better understanding of the natural history is crucial to rigorous therapeutic trials. Methods: A cohort of 83 patients with Becker muscular dystrophy (5–75 years at baseline) were followed for up to 3 years with annual assessments. Muscle and pulmonary function outcomes were analyzed herein. Age-stratified statistical analysis and modeling were conducted to analyze cross-sectional data, time-to-event data, and longitudinal data to characterize these clinical outcomes. Results: Deletion mutations of dystrophin exons 45–47 or 45–48 were most common. Subgroup analysis showed greater pairwise association between motor outcomes at baseline than association between these outcomes and age. Stronger correlations between outcomes for adults than for those under 18 years were also observed. Using cross-sectional binning analysis, a ceiling effect was seen for North Star Ambulatory Assessment but not for other functional outcomes. Longitudinal analysis showed a decline in percentage predicted forced vital capacity over the life span. There was relative stability or improved median function for motor functional outcomes through childhood and adolescence and decreasing function with age thereafter. Conclusions: There is variable progression of outcomes resulting in significant heterogeneity of the clinical phenotype of Becker muscular dystrophy. Disease progression is largely manifest in adulthood. There are implications for clinical trial design revealed by this longitudinal analysis of a Becker natural history dataset.

show abstract

“…Onset is usually between the ages of 5 and 15 years, but may be later, and the majority of patients are still able to walk when they are 20. The disease progresses more slowly and more gently than does Duchenne MD (the onset of which is in early childhood) (Griggs et al 1995, Harper 1985, Kakulas 1999, McDonald et al 1995, Walton & Gardner-Medwin 1988.…”

Section: Some Of the Diagnoses Included In The Proximal Muscular Dystmentioning

confidence: 99%

Living with Muscular Dystrophy: Illness Experience, Activities of Daily Living, Coping, Quality of Life and Rehabilitation

Nätterlund

2001

Scandinavian Journal of Occupational Therapy

View full text Add to dashboard Cite

Becker??s Muscular Dystrophy

Cited by 62 publications

References 0 publications

Dystrophic muscle distribution in late-stage muscular dystrophy

Dystrophic muscle distribution in late-stage muscular dystrophy

Findings from the Longitudinal CINRG Becker Natural History Study

Living with Muscular Dystrophy: Illness Experience, Activities of Daily Living, Coping, Quality of Life and Rehabilitation

Contact Info

Product

Resources

About