Review of Phase II and Phase III clinical trials for Duchenne muscular dystrophy

Abstract

Introduction: Evidence-based therapeutics in Duchenne muscular dystrophy (DMD) has been limited to corticosteroids for the past 30 years. There have been a host of other therapeutic interventions studied in mice, canines and more recently humans, but they are yet to show effectiveness in clinical trials. Newer genetic approaches are in early stages of clinical trials.

Areas covered: In this paper, the authors review evidence-based studies for corticosteroids as well as other Phase II and Phase III clinical trials involving potential pharmacologic treatments: myostatin and phosphodiesterase inhibitors, insulin-like growth factor 1 and replenishment of nutritional deficiencies. Finally, the authors briefly review the current status of treatments specific for genetic mutations and gene therapy.

Expert opinion: Since the identification of corticosteroids as an effective treatment for DMD, there has not yet been another pharmacologic intervention that has shown as much benefit, although further investigation is needed for some of the mentioned therapeutics. New therapies will need to show a significantly greater sustained benefit for our DMD patients with cost effectiveness, in order for them to supplant or reduce the use of long-term corticosteroid treatment. While studying new therapeutics, further study and trials in corticosteroids should not be lost.

Keywords::

• clinical trials
• corticosteroids
• Duchenne muscular dystrophy
• therapeutics

Notes

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