Dantrolene: An Exon Skipping Enhancing Drug
Drs. Carrie Miceli and Stan Nelson at UCLA are developing an FDA approved drug that could boost the effectiveness of exon skipping drugs that are being developed and tested for Duchenne muscular dystrophy.
Drs. Miceli and Nelson in collaboration with Dr. Melissa Spencer, have tested dantrolene in combination with morpholinos in the mdx mouse, the mouse model for Duchenne. The increase in effectiveness is significant given the difficulty and high price of producing morpholinos. Any reduction in the amount of morpholinos that need to be administered will greatly benefit the Duchenne patients and community.