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Duchenne Muscular Dystrophy
Duchenne muscular dystrophy (DMD) is a genetic disorder caused by a change in the DMD gene. This disorder causes muscles to break down and become weaker over time. It is an inherited condition that parents pass to children.
DMD affects mostly boys. It occurs in about 1 in 3,500 to 5,000 babies from all ethnic groups.
While there is no cure for DMD, genetic treatments are being tested. Some of these treatments are available to certain people with DMD. Your child’s care team will work to manage symptoms and help your child maintain function.
Duchenne muscular dystrophy is caused by a gene change, called a mutation. A change in the DMD gene affects a protein made by muscle cells called dystrophin. When the gene is changed or deleted, muscles do not produce enough dystrophin. Without that protein, muscle fibers do not work as they should. This leads to muscle damage, muscle breakdown, and weakness.
The DMD gene is located on the X chromosome. A chromosome is the part of a cell that contains genetic information.
Girls have 2 X chromosomes, 1 from their mother and 1 from their father. Because girls have 2, they can usually make enough dystrophin with the gene from the healthy X chromosome. Girls with this gene change may have some symptoms, such as weakness, heart problems, and muscle cramps. These girls are also carriers of the disease. They can pass it on to their children.
Boys have an X chromosome from their mother and a Y chromosome from their father. If the gene for dystrophin on their X chromosome is affected, they cannot create more dystrophin. Boys with DMD have more severe symptoms that get worse over time and have a shorter life expectancy.
To learn more about DMD and current treatments, visit organizations dedicated to DMD and rare diseases.
Duchenne muscular dystrophy care at St. Jude
St. Jude Children’s Research Hospital is working to deepen the understanding of rare, catastrophic genetic neurological disorders in children. Our aim is to transform the treatment landscape for these disorders. The Center for Experimental Neurotherapeutics (CENT) was established at St. Jude as part of the Pediatric Translational Neuroscience Initiative (PTNI) to focus on these neurological disorders.
Select children with DMD who are accepted at St. Jude take part in clinical research or clinical trials. The aim of these studies is to help scientists learn more about the disorders and to develop potential treatments.
Duchenne muscular dystrophy clinical research
Recruiting
Study goal:
The main goal of this study is to see if DYNE-251 is safe to use in young males with DMD. Researchers also want to find out how well DYNE-251 treats the disease and how it affects the body in other ways.
Age:
Male, age 4 to 16 years old
Seeking treatment at St. Jude
Patients accepted to St. Jude must have a disease we treat and must be referred by a physician or other qualified medical professional. We accept most patients based on their ability to enroll in an open clinical trial.
Contact the Physician / Patient Referral Office
Call: 1-888-226-4343 (toll-free) or 901-595-4055 (local) | Fax: 901-595-4011 | Email: referralinfo@stjude.org | 24-hour pager: 1-800-349-4334