Summary

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• This summary is based on the review of one systematic review/meta-analysis. ^[1]
• Golodirsen (Vyondys 53) is conditionally approved for treating Duchenne muscular dystrophy (DMD) by targeting specific mutations in the DMD gene, similar to other drugs like eteplirsen and ataluren, which also address the underlying genetic cause of the disease.
• One study notes that golodirsen, eteplirsen, and ataluren are effective for small sub-populations with specific genetic mutations; however, it does not provide comparative effectiveness data among these drugs or details on differences in effectiveness across various population types or subgroups.
• It is highlighted that while some experimental therapies have shown promising effects in skeletal muscle, they are largely ineffective in the cardiac system, an important consideration for DMD patients who often experience cardiac and respiratory complications.
• There is no safety information available in the reviewed studies.