New and emerging pharmacotherapy for duchenne muscular dystrophy: a focus on synthetic therapeutics
ABSTRACT Introduction Duchenne muscular dystrophy (DMD) is the result of X chromosome linked mutations to the dystrophin protein gene that prevent the normal development and repair of muscles leading to muscle deterioration. The condition affects nearly 1 in 3,500 males worldwide. Current therapeutics have not been sufficient in providing a cure or resulting in a significant extension in life expectancy, but many therapeutic options are currently under investigation. Areas covered This article provides an overview of the current and emerging therapies for DMD giving particular focus to synthetic therapeutic options. The authors further provide their expert opinion. Expert opinion Many discrepancies in primary outcomes of trials have led to questions of efficacy for medications, as well as difficulty in securing FDA approval. A standardization of primary outcome strategies, as well as better access to investigational medications, may alleviate some of the controversy and pressures that exist on medication approvals. Many trials have identified cohorts who responded more favorably to medications, despite a lack of significance in the overall intent to treat populations. This indicates that more medication screening and personalized treatment with patient specific targeting might deliver more clinically significant results.
Expert opinion on pharmacotherapy