Dr. Jerry Mendell of Nationwide Children’s Research Institute recently presented data from a ground-breaking PPMD-funded study demonstrating that a modified virus carrying the gene for follistatin can improve performance on the 6 minute walk test in study participants with Becker muscular dystrophy. This is the first time a gene…

Late Breaking Talks:

PPMD-funded gene therapy study improves walking ability in muscular dystrophy

Dr. Jerry Mendell of Nationwide Children’s Research Institute presented data from a ground-breaking PPMD-funded study demonstrating that a modified virus carrying the gene for…

PTC Therapeutics Symposium – “Improving Outcomes for the Future: Making sense of the nonsense mutations in Duchenne muscular dystrophy”

PTC Therapeutics gave an overview of the data supporting the recent accelerated approval for Translarna® in Europe and answered questions from…

Sarepta held a symposium on the role of dystrophin production in disease-modifying treatments for Duchenne. Some highlights:

We heard from Dr. Francesco Muntoni from the Dubowitz Neuromuscular Centre about how much dystrophin is expected to be needed to stabilize the muscles and result…