Duchenne muscular dystrophy (DMD)is a severe, progressiveneuromuscular diseasethat affects the muscles.
How Common Is DMD?
Duchenne muscular dystrophy is a rare disease, affecting roughly 1 out of every 3,600 to 6,000 men.
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Few womenabout 1 in 50,000develop active forms of the disease.
There is no cure for DMD.
Many people with the condition do not live beyond their 30s.
Treatment has come a long way, though, adding years to the lives of people with DMD.
This article will explore how DMD develops, what symptoms to expect, and how the condition is treated.
The earliest symptoms can include waddling, difficulty walking, and frequent falling.
As the condition progresses, many children with DMD will become dependent on a wheelchair.
This usually occurs by around the age of 10.
Many people with DMD die fromcardiacorrespiratory failure.
Causes
DMD is a condition that you are born with.
The genetic pattern means that DMD is more common in boys than in girls.
Men cannot pass DMD on to their sons, but their daughters will be carriers of the mutation.
If you have a known family history of DMD,genetic testingmay have already been done.
There will also be a range of healthcare providers involved in your care.
People who get optimal care have had years added to their lives.
Treatment Goals
There is no cure for DMD.
DMD can also be challenging for the family and friends of someone with the condition.
Summary
Duchenne muscular dystrophy is a progressive, genetic condition that mostly affects men.
While women can inherit the gene, they are usually only carriers and do not have active symptoms.
Over time, DMD causes muscle weakness and eventually muscle loss.
Early on, a person will have motor delays and falls.
Frequently Asked Questions
Yes.
The genetic mutation that causes DMD is passed through families.
There is no cure for DMD, but the life expectancy for people with this condition has increased.
Ongoing clinical trials and new medications are being investigated to help slow the progression of the disease.
Once the muscles in the heart and the lungs are affected, DMD usually causes fatal complications.
Few people with DMD live beyond their 30s.
Muscular Dystrophy Association.Duchenne muscular dystrophy (DMD).
National Institutes of Health.Duchenne muscular dystrophy.
Updated November 2, 2021.
National Institutes of Health, Genetic and Rare Diseases Information Center.Duchenne muscular dystrophy.
Updated November 20, 2020.
National Institutes of Health.DMD gene.
Updated August 18, 2020.
Min YL, et al.CRISP correction of Duchenne muscular dystrophy.Ann Rev Med.
January 2019;70:239-255. doi:10.1146/annurev-med-081117-010451.
U.S. Food and Drug Administration.FDA approves targeted treatment for rare Duchenne muscular dystrophy mutation.
Updated February 25, 2021.
Verhaart, I.E.C., Aartsma-Rus, A.Therapeutic developments for Duchenne muscular dystrophy.Nat Rev Neurol.July 2019.
