Investigational treatments and therapeutic targets in Becker muscular dystrophy

Abstract

Introduction: Becker muscular dystrophy (BMD) is a progressive X-linked muscle-wasting disease caused by mutations in the dystrophin gene. Duchenne muscular dystrophy (DMD) is also caused by such mutations but shows rapidly progressive muscle wasting. Therefore, studies aimed at establishing treatments have been focused on DMD. Fortunately, most DMD treatments can also be applied as BMD treatments because of the common pathophysiology. Although the natural history of BMD is very heterogeneous, this is a debilitating disease with progressive muscle weakness. Thus, treatments for BMD are urgently needed.

Areas covered: Treatments for BMD are described by reviewing treatments for DMD, as BMD and DMD share a common pathophysiology caused by dystrophin abnormalities. Additionally, the characteristics of BMD patients aged over 60 years are summarized.

Expert opinion: Until now, many efforts have been made to establish treatments for DMD, while efforts toward BMD treatments have not been prominent. It is now time to make more effort to establish BMD treatments, especially with regard to modulation of splicing in BMD-specific mutations. Although most DMD treatments can be applied as BMD treatments, it must be remembered that some BMD patients are able to live a near-normal life even with a mutation in the dystrophin gene.

Keywords:

• Becker muscular dystrophy
• Duchenne muscular dystrophy
• splicing modulation
• treatment

Declaration of interest

The author has no relevant affiliations or financial involvement with any organization or entity with a financial interest in or financial conflict with the subject matter or materials discussed in the manuscript. This includes employment, consultancies, honoraria, stock ownership or options, expert testimony, grants or patents received or pending, or royalties.

Notes

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