Trial Design and Outcome Measurement in Muscular Dystrophies

Abstract

Advances in genetics have resulted in the development of several disease modifying treatments for muscular dystrophies. These drugs have to be tested for efficacy before they can be used in clinical practice. The design and conduct of clinical trials for muscular dystrophies offers several challenges because of the rarity of these disorders, the need for sensitive and responsive clinical outcome measures and for surrogate biomarkers. Innovations such as digital health technologies and adaptive trial platforms offer options for decentralized, quicker and less expensive trials.