Drug development for rare mitochondrial disorders.

ABSTRACT: Currently, all treatment of mitochondrial disorders is performed with dietary supplements or by off-label use of drugs approved for other indications. The present challenge is translation of our collective knowledge of the molecular details underlying the pathophysiology of mitochondrial disorders into safe and effective therapies that are approved by the regulatory authorities. Molecular details permit precise diagnoses, but homogeneity is gained at the expense of limiting numbers of subjects for clinical trials and of small markets from which to recoup the considerable expense of drug discovery and development. The Food and Drug Administration recognizes that trial designs suitable for common diseases are often not feasible for rare disorders. They have developed a number of programs to

SUBMITTER: Hurko O 

PROVIDER: S-EPMC3625383 | biostudies-literature | 2013 Apr

REPOSITORIES: biostudies-literature