Developing DMD therapeutics: a review of the effectiveness of corticosteroids, stop-codon readthrough, and exon-skipping therapies.

Developing DMD therapeutics: a review of the effectiveness of corticosteroids, stop-codon readthrough, and exon-skipping therapies.
Expert Opin Investig Drugs. 2021 Jan 04;:
Authors: Sheikh O, Yokota T
Abstract
INTRODUCTION: Duchenne muscular dystrophy (DMD) is an X-linked recessive disorder caused by mutations in the dystrophin (DMD) gene. Most patients die from respiratory failure or cardiomyopathy. There are significant unmet needs for treatments for DMD because the standard of care is principally limited to symptom relief through treatments such as steroids.
AREAS COVERED: This review summarizes safety and efficacy in promising areas of DMD therapeutics - steroids, stop codon readthrough, gene replacement therapy, and exon-skipping - under clinical examination from 2015-2020 as demonstrated in the NIH Clinical Trials and PubMed search engines. Furthermore, we discuss therapy effectiveness, current challenges such as regulatory hurdles, and future perspectives.
EXPERT OPINION: Currently, steroids persist as the most accessible medicine for DMD. Stop codon readthrough, gene replacement therapy, and exon-skipping therapy all aim to restore dystrophin expression. Of these strategies, gene replacement therapy has recently gained momentum while exon-skipping retains great traction. The clinical progress of viltolarsen and FDA approval of eteplirsen and golodirsen illustrate this regulatory momentum, though the effective...

Source: Expert Opinion on Investigational Drugs - January 5, 2021 Category: Drugs & Pharmacology Tags: Expert Opin Investig Drugs Source Type: research