Therapeutic trials and muscular diseases]

Recent advances in knowledge of muscle diseases has opened new perspectives for clinical trials. Because of the small number of patients available for study and the requirement for homogeneous study groups, multicentric trials must be conducted on a national or international basis. Moreover, because of the variability of clinical course of muscle diseases therapeutic protocols must be preceded by an assessment of the natural history of the disease. Analytic methods available include manual testing, quantitative muscle testing (QMT), functional scales, and evaluation of cardiac and respiratory function. Based on experience acquired with earlier protocols, we present methodologies currently used and the difficulties encountered. We discuss the potential ways for improving therapeutic trials.