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Carvedilol can prevent cardiac events in Duchenne muscular dystrophy
Tsuyoshi Matsumura1, Takuhisa Tamura, Satoshi Kuru
1Department of Neurology, National Hospital Organization Toneyama National Hospital, Toyonaka, Japan. matumura@toneyama.hosp.go.jp
Insights
Beta-blocker medication, specifically carvedilol, shows promise in preventing cardiac events in Duchenne muscular dystrophy patients. This study suggests carvedilol is safe and effective for managing heart failure in this population.
Area of Science:
- Cardiology
- Neuromuscular Disorders
- Pharmacology
Background:
- Heart failure is a critical complication in Duchenne muscular dystrophy (DMD).
- Beta-blockers improve outcomes in adult heart failure, but their use in DMD is not well-established.
- Assessing carvedilol's efficacy and safety in DMD patients is crucial.
Purpose of the Study:
- To evaluate the efficacy and safety of carvedilol in DMD patients with heart failure.
- To compare cardiac event rates and survival in DMD patients with and without carvedilol treatment.
Main Methods:
- A multicenter open trial involving 54 DMD patients.
- 41 patients received carvedilol (BB group), 13 did not (non-BB group).
- Cardiac function and clinical signs were monitored; patients with ejection fraction <50% received ACE inhibitors.
Main Results:
- The carvedilol group had a higher survival rate free from primary endpoints (death, heart failure deterioration, severe arrhythmia).
- Higher mean heart rates and lower carvedilol doses were observed in patients experiencing primary endpoints.
- Carvedilol introduction was generally safe, though patients with advanced dysfunction required longer titration and experienced minor complaints.
Conclusions:
- Carvedilol appears to be relatively safe for DMD patients.
- Carvedilol may help prevent cardiac events in DMD patients.
- Further research may support carvedilol as a standard treatment for DMD-associated heart failure.
Objective:
Heart failure is one of the most serious complications in Duchenne muscular dystrophy (DMD). Beta-blocker medication is known to improve the prognosis of chronic heart failure of adults, but its efficacy and safety for DMD patients has not been fully assessed. Thus we conducted a multicenter open trial.
Methods:
Fifty-four DMD patients participated; 41 received carvedilol (BB group) and 13 did not (non BB group). All patients with an ejection fraction of less than 50% received angiotensin-converting enzyme inhibitor. Then, patients in BB group were started on carvedilol. The mean maintenance dose of carvedilol in BB group was 7.85+/-2.80 mg/day. Clinical signs and cardiac function were monitored regularly and statistical analysis was done.
Results:
The survival rate free from primary endpoints (death, deterioration of heart failure and severe arrhythmia) was higher in the BB group. The survival rate free from all-cause death was also higher in the BB group, although not significantly higher. Patients with primary endpoints received lower maintenance doses of carvedilol and presented higher mean heart rates (HR) during the observation period. In the BB group, mean HR at enrollment and the reduction of mean HR were correlated with the change of ejection fraction. Although serious adverse events were rare during the introduction of carvedilol, patients with advanced cardiac dysfunction required a longer period for up-titration and frequently presented with minor complaints.
Conclusion:
The present study suggests that carvedilol is relatively safe and can prevent cardiac events even in patients with DMD.
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