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Duchenne muscular dystrophy

  • About
  • About
Overview
Symptoms
Causes
Types
  • Duchenne muscular dystrophy
  • Other cardio-skeletal myopathies and dystrophies
Diagnosis
Treatment
Overview
Symptoms
Causes
Types
  • Duchenne muscular dystrophy
  • Other cardio-skeletal myopathies and dystrophies
Diagnosis
Treatment

Duchenne Muscular Dystrophy

Duchenne muscular dystrophy is the most common limb girdle muscular dystrophy. The gene that is mutated in Duchenne muscular dystrophy, DMD, encodes dystrophin. DMD is encoded in the DNA of the X chromosome. The X chromosome is one of two sex chromosomes, X and Y. Males, who have one X chromosome, are much more likely to develop Duchenne muscular dystrophy; while females are usually carriers of the DMD mutation in families with Duchenne muscular dystrophy, and may have no clinical phenotype. 

Boys with Duchenne muscular dystrophy typically have onset of disease in childhood and progressive symptoms of skeletal muscle weakness through the teenage years and early adulthood. Patients become wheelchair bound and develop respiratory muscle weakness. As dystrophin is also important for heart muscle function, the heart can become weakened and develop arrhythmias due to the dystrophin mutation.

Treatment of Duchenne muscular dystrophy

Treatment with medications used for dilated cardiomyopathy and heart failure can prevent worsening of heart muscle dysfunction and reduce heart-related symptoms.

Learn more about duchenne muscular dystrophy.

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Clinical Trials

Clinical trials are research studies that evaluate a new medical approach, device, drug, or other treatment. As a Stanford Health Care patient, you may have access to the latest, advanced clinical trials.

Open trials refer to studies currently accepting participants. Closed trials are not currently enrolling, but may open in the future.

Open Trials Closed Trials
Clinical Trials View All »
A Clinical Study to Assess Two Doses of GSK2402968 in Subjects With Duchenne Muscular Dystrophy (DMD) Duchenne Muscular Dystrophy (DMD), 
An Open-Label, Multi-Center, 48-Week Study With a Concurrent Untreated Control Arm to Evaluate the Efficacy and Safety of Eteplirsen in Duchenne Muscular Dystrophy Muscular Dystrophy
Drisapersen Duchenne Muscular Dystrophy (DMD) Treatment Protocol Muscular Dystrophy, Duchenne Muscular Dystrophy (DMD), 
CHAR0312 DMD tissue bank for Exon skipping Muscular Dystrophy, Duchenne muscular dystrophy

Clinics for Duchenne muscular dystrophy

  • Neuromuscular Program
    650-723-6469
  • Center for Inherited Cardiovascular Disease
    650-736-7878
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