Introduction: Duchenne muscular dystrophy (DMD) is an incurable neuromuscular disorder of childhood. Healthcare, caregiving, and other resource needs of affected individuals are thought to be substantial; however, the economic burden associated with DMD has not yet been assessed specifically in Australia.

Methods: Australian households with a child with DMD were asked to complete a cross-sectional survey. Data were collected on annual resource utilization including hospital and medical services, equipment, home modifications, informal care, and working days lost.

Results: Mean healthcare costs were found to be $10,046 Australian dollars per affected individual and were markedly higher than average Australian health expenditures at each age group. The mean total cost was $46,700 (median $32,300), with healthcare costs contributing 22% of total costs.

Conclusions: The annual economic cost of DMD was found to be high, reflecting a significant socioeconomic burden, especially in boys who reach adulthood, where household resource use and caregiving burden is highest. Muscle Nerve 53: 877-884, 2016.

Download full-text PDF

Source
http://dx.doi.org/10.1002/mus.24965DOI Listing

Publication Analysis

Top Keywords

duchenne muscular
8
muscular dystrophy
8
healthcare costs
8
health care
4
care utilization
4
utilization costs
4
costs children
4
children adults
4
adults duchenne
4
dystrophy introduction
4

Similar Publications

Non-invasive pressure-volume loop derived temporal elastance, contractility, and efficiency indices for assessing Duchenne muscular dystrophy patients.

Heart Vessels

December 2024

Department of Biomedical Engineering, Veterans Affairs Medical Center, University of Cincinnati, Rhodes Hall 593, 2851 Woodside Drive, Cincinnati, OH, 45219, USA.

Ejection fraction is commonly used to assess Duchenne muscular dystrophy-associated cardiomyopathy (DMDAC), but it may remain normal (wrongly) despite significant myocardial dysfunction in patients. Therefore, better indicators of myocardial dysfunction are needed for longitudinal (with time) assessment and treatment of DMDAC patients. This study evaluates non-invasive LV PV loop-derived elastance, contractility and efficiency in relation to EF for patients developing DMDAC.

View Article and Find Full Text PDF

The Potential of Targeting APE1/Ref-1 as a Therapeutic Intervention for Duchenne Muscular Dystrophy.

Antioxid Redox Signal

December 2024

Department of Medicine-Western Health, Melbourne Medical School, The University of Melbourne, Parkville, Australia.

Inflammation and oxidative stress play crucial roles in the development and progression of skeletal muscle diseases. This review aims to examine the existing evidence regarding the involvement and inhibition of APE1/Ref-1 (apurinic/apyrimidinic endonuclease 1/redox factor 1) in diseases, then extrapolate this evidence to the context of skeletal muscle and discuss the potential beneficial effects of APE1/Ref-1 inhibition in ameliorating myopathy with a particular focus on dystrophic pathology. Currently, therapeutic interventions targeting pathways, such as nuclear factor kappa-light-chain-enhancer of activated B cells (NF-κB) and nuclear factor erythroid 2-related factor 2 (NRF2), have shown limited efficacy in both clinical and preclinical settings.

View Article and Find Full Text PDF

Intrinsic Muscle Stem Cell Dysfunction Contributes to Impaired Regeneration in the mdx Mouse.

J Cachexia Sarcopenia Muscle

February 2025

Sprott Centre for Stem Cell Research, Regenerative Medicine Program, Ottawa Hospital Research Institute, Ottawa, Canada.

Background: Duchenne muscular dystrophy (DMD) is a devastating disease characterized by progressive muscle wasting that leads to diminished lifespan. In addition to the inherent weakness of dystrophin-deficient muscle, the dysfunction of resident muscle stem cells (MuSC) significantly contributes to disease progression.

Methods: Using the mdx mouse model of DMD, we performed an in-depth characterization of disease progression and MuSC function in dystrophin-deficient skeletal muscle using immunohistology, isometric force measurements, transcriptomic analysis and transplantation assays.

View Article and Find Full Text PDF

Introduction: Duchenne muscular dystrophy (DMD) is a severe X-linked disorder characterized by progressive muscle weakness and eventual death due to cardiomyopathy or respiratory complications. Currently, there is no cure for DMD, with standard treatments primarily focusing on symptom management. Using immunosuppressive measures and optimized vector designs allow for gene therapies to better address the underlying genetic cause of the disease.

View Article and Find Full Text PDF

Patient- and Caregiver-Reported Impact of Symptoms in Alzheimer Disease, Mild Cognitive Impairment, and Dementia.

Neurol Clin Pract

February 2025

University of Rochester School of Medicine and Dentistry (JS, AV); Center for Health and Technology (CHeT) (JS, JW, AV, SJR, CE, AA, CZ, CRH), University of Rochester; University of Utah Spencer Fox Eccles School of Medicine (SJR); Des Moines University College of Osteopathic Medicine (AA); Department of Biostatistics and Neurology (ND), University of Rochester; Alzheimer's Disease Care, Research and Education Program (AD-CARE) (AM, SS-S, EJS), University of Rochester; and Department of Neurology (CRH), University of Rochester.

Background And Objectives: In preparation for future clinical trials involving individuals with Alzheimer disease (AD), mild cognitive impairment (MCI), and dementia, it is important to ascertain the widespread impact of symptoms from the direct perspectives of patients and caregivers. In this study, we performed cross-sectional surveys using large-scale patient and caregiver data to identify the prevalence and average impact of symptoms and symptomatic themes experienced by adults with AD, MCI, and dementia. Subsequent analyses were used to determine which demographic and disease-specific factors are associated with more severe disease.

View Article and Find Full Text PDF

Want AI Summaries of new PubMed Abstracts delivered to your In-box?

Enter search terms and have AI summaries delivered each week - change queries or unsubscribe any time!