Specialties & Conditions Duchenne Muscular Dystrophy
Duchenne Muscular Dystrophy
Program
Duchenne muscular dystrophy is a form of muscular dystrophy, an inherited disease which occurs in about 1 out of every 3,600 male infants. It worsens more quickly than other types of muscular dystrophy such as Becker's muscular dystrophy.
Symptoms may include fatigue, learning difficulties (the IQ can be below 75), intellectual disability (possible). Muscle weakness begins in the legs and pelvis but also occurs less severely in the arms, neck, and other areas of the body.
Problems with motor skills develop, such as with running, hopping, and jumping. Frequent falls occur, as well as trouble getting up or with climbing stairs. Weakness quickly gets worse.
The ability to walk may be lost by age 12, and a wheelchair will become necessary. Finally, breathing difficulties and heart disease begin, usually by age 20.
Duchenne muscular dystrophy progressively worsens, and death often occurs by age 25, typically from lung disorders. However, advances have resulted in many men living much longer.
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Related News
With Comprehensive Cardiology Care, Xavier Kicks Off a Bright Future
Xavier received a life-saving heart transplant, becoming the first patient with muscular dystrophy to undergo this surgery at Lurie Children's. His strength helps pave the way for future heart transplants in children with neuromuscular disorders.
Lurie Children’s First in Illinois to Treat Duchenne Muscular Dystrophy with FDA-Approved Gene Therapy
In March, Lurie Children’s treated its first patient with ELEVIDYS, the first gene therapy for Duchenne muscular dystrophy – a rare, genetic disease characterized by progressive muscle damage and weakness. Lurie Children’s is the first in Illinois to administer this treatment after ELEVIDYS received FDA approval in 2023.
Novel Drug May Improve Treatment for Duchenne Muscular Dystrophy
NS Pharma Announces Publication of Clinical Trial Data for Viltolarsen in DMD Patients in JAMA Neurology
