New findings raise questions on process to identify R&D drugs for rare diseases

A study by US National Institutes of Health researchers has revealed surprising new insights into the process used to initially identify an  experimental drug now being tested in people with cystic fibrosis and  muscular dystrophy. Researchers emphasized the clinical implications of  their findings are unclear, but said the results suggest more work may  be needed to make sure the screening process to select promising agents  was not flawed by its effects on a firefly enzyme used as a marker. The  study was published this month in the Proceedings of the National  Academy of Sciences.