FSHD Facioscapulohumeral Muscular Dystrophy: Clinical Medicine and Molecular Cell Biology, edited by Meena Upadhyaya and David N.Cooper. © 2004 Garland/BIOS Scientific Publishers Limited, Abingdon.

24.1 Introduction

The explosion of molecular genetic information has provided tangible new targets for therapeutic interventions in a number of muscular dystrophies. Yet, despite this wealth of genetic discoveries, no new effective therapeutic interventions exist for the treatment of muscular dystrophies. Moreover, the enticing promise, yet to be fulfilled, of gene therapy has led to the relative neglect of research into potential pharmacologic interventions.