Improving clinical trial design for Duchenne muscular dystrophy.

ABSTRACT:

Background

Currently, the most promising therapies for Duchenne muscular dystrophy (DMD) are exon skipping and stop codon read-through, two strategies aimed at restoring the expression of dystrophin. A phase 3 clinical trial with drisapersen, a drug designed to induce exon 51-skipping, has failed to show significant improvement of the primary outcome measure, the six-minute walk test.

Discussion

Here, we review some key points that should be considered when designing clinical trials for these new therapies. First, younger patients have more functional abilities and more muscle fibers to preserve than older patients and therefore are better subjects for trials designed to demonstrate the success of new treatments. Second, the inclusion of patients on corticosteroids both in t

SUBMITTER: Merlini L 

PROVIDER: S-EPMC4549867 | biostudies-literature | 2015 Aug

REPOSITORIES: biostudies-literature