Dr. Bönnemann, Dr. Dowling, Drug, Muscular Disorder, National Institutes of Health, Pediatric, Rare Disease, The Hospital for Sick Children
Repurposing a Cancer Drug to Treat a Rare Disease Principal Investigator: Dr. James Dowling and Dr. Carsten Bönnemann Disease: Myotubular Myopathy Research Description: X-linked myotubular myopathy (MTM) is a rare disorder that affects males and is caused by mutations...
