Duchenne Muscular Dystrophy Care Considerations
Duchenne muscular dystrophy (DMD) is a rare genetic (inherited) disease defined by muscle weakness that gets worse over time and ultimately affects the heart and lungs. People born with DMD will see many healthcare providers throughout their lives.
The updated care considerations for DMD are contained in a set of three articles published in Lancet Neurology. They offer the latest clinical considerations to improve care and quality of life for people living with DMD. Clinicians who specialize in treating people with DMD developed these care considerations based on current evidence for optimal care reported in the literature and their own clinical experiences.
As science and medicine are advancing, people with DMD are living longer; therefore, their care throughout life is evolving as well. These care considerations, funded by the Centers for Disease Control and Prevention (CDC), are intended to raise the standards of care, help clinicians provide the best possible care to people with DMD, and give families and caregivers the necessary information to manage their care.
Read the articles here:
- Diagnosis and management of Duchenne muscular dystrophy, part 1: Diagnosis, neuromuscular, rehabilitation, endocrine, and gastrointestinal and nutritional managementExternal
- Diagnosis and management of Duchenne muscular dystrophy, part 2: Respiratory, cardiac, bone health, and orthopaedic managementExternal
- Diagnosis and management of Duchenne muscular dystrophy, part 3: Primary care, emergency management, psychosocial care, and transitions of care across the lifespanExternal
Care Considerations Pediatrics Supplement
The Pediatrics supplement, also funded by the CDC, is a set of 13 articles that expands on the clinical care for each subspecialty outlined in the Lancet Neurology articles. The supplement provides new resources, including a toolkit to help individuals with DMD transition into adult medical care. These articles and resources can help families and providers manage DMD care. Also included is an article on evaluation of the care considerations, with key elements of care that clinics can measure.
Resources Available to People with DMD and Their Families
The DMD Family Guide Cdc-pdf[PDF – 6.66 MB]External, created by a group of non-profit organizations that work to improve the lives of people with DMD and their families, summarizes the content of the care considerations articles in a printable-booklet format. This guide is designed to give families detailed information on the medical care that individuals with DMD need at each stage of their lives.
Parent Project Muscular Dystrophy has several resourcesExternal available for people with DMD and their families, including web information about specific care considerations topics.
CDC’s Work on Duchenne Muscular Dystrophy
CDC’s National Center on Birth Defects and Developmental Disabilities has been working on projects aimed at improving the diagnosis and care of people with DMD for over 15 years. Some of these activities include the following:
Muscular Dystrophy Surveillance, Tracking, and Research Network (MD STARnet) collects health information on people with muscular dystrophy in specific areas of the United States. Contributions from MD STARnet to DMD research include generating information on delays in diagnosis, treatment methods, prevalence, types of care received, growth patterns of people with DMD, and others. Read more articles from MD STARnet on Duchenne and Becker muscular dystrophies
Physical Developmental Delays: What to look forExternal is a free online tool that helps parents determine when to be concerned about the physical development of their child and to notify their doctor.
Child Muscle WeaknessExternal is a free online tool designed to help clinicians, therapists, and other specialists diagnose muscle weakness in children as early as possible. Early diagnosis could lead to more personalized care for each person living with muscular dystrophy and may give each of them a better chance to reach his or her full potential.
CDC has funded studies and worked with partners on newborn screening of DMD:
Evidence-based Path to Newborn Screening for Duchenne Muscular DystrophyExternal
Annals of Neurology, 2012 Mar;71(3):304-13
Mendell JR, Shilling C, Leslie ND, et al.
Clinical Follow-Up for Duchenne Muscular Dystrophy Newborn Screening: A ProposalExternal
Muscle & Nerve, 2016 Aug;54(2):186-91
Kwon JM, Abdel-Hamid HZ, Al-Zaidy SA, et al.
CDC also funded the original DMD Care Considerations articles, published in 2010 by Lancet Neurology.
- Read more about care considerations for DMD
- Read about the MD CARE ActExternalExternal, which authorizes the CDC to work on muscular dystrophy activities
- Parent Project Muscular Dystrophy (PPMD)ExternalExternal
A non-profit parent advocacy group focusing on DMD. PPMD funds research, raises awareness, promotes advocacy, makes connections within the DMD community, and broadens treatment options.
- Muscular Dystrophy Association (MDA)ExternalExternal
A national non-profit agency that works to cure muscular dystrophy through research. MDA provides health care, support services, advocacy, and education.