Early study shows Dantrium (JHP Pharmaceuticals) has promise for treating Duchenne Muscular Dystrophy

Scientists at UCLA have found Dantrium (sodium dantrolene), from JHP Pharmaceuticals, provides a powerful boost to drug therapy for Duchenne Muscular Dystrophy. The hope is that when used in combination, the drug will provide a one-two punch that will overcome the genetic mutations that cause Duchenne Muscular Dystrophy, and restore a missing protein needed for proper muscle function, and allow those affected by the disease to lead relatively normal lives.

The results of thie early study are published in Science Translational Medicine.

Dantrolene was found using a high-throughput molecular screening technique that allows users to scrutinize many molecules at the same time, according to the study's senior authors, Dr. Stanley Nelson, a UCLA professor of human genetics, and Carrie Miceli, a UCLA professor of microbiology, immunology and molecular genetics. The drug is already approved in the US and EU as a muscle relaxant for those with conditions such as cerebral palsy, multiple sclerosis, stroke, or spinal cord injury. See: "Dantrolene enhances antisense-mediated exon skipping in human and mouse models of duchenne muscular dystrophy." Kendall GC et al. Sci Transl Med. 2012 Dec 12;4(164):164ra160. doi: 10.1126/scitranslmed.3005054.