IntroductionDuchenne muscular dystrophy (DMD) is a progressive genetic disease leading
to muscular weakness. DMD is caused by mutations of the dystrophin gene on the X chromosome
that is responsible for production of dystrophin protein. Dystrophin contributes to
structural support in muscle cells and mutations result in dystrophin protein deficiency
which causes muscle damage and the associated clinical presentation. Exon skipping
medications, including the exon 53 targeting viltolarsen, are the first agents with
the ability to partially restore dystrophin protein.Areas coveredHerein, the authors
profile viltolarsen for the DMD patients who are amenable to exon 53 skipping therapy
and provide their expert perspectives on this subject.Expert opinionCurrent findings
suggest that viltolarsen could play a role in the current and possible future treatment
of DMD. Viltolarsen seems to be safe and restores dystrophin protein to around 6%
of the normal level. Due to orphan drug status, after the completion of the phase
2 clinical trial, viltolarsen was granted accelerated approval in Japan and in the
US. A phase 3 trial is currently in progress and needs to earn full approval. Although
a multidisciplinary approach continues to be critical, the addition of exon skipping
agents like viltolarsen may improve the quality of patients' lives. However, data
on the long-term safety and efficacy of this medication are not yet available due
to its recent accelerated approval.
