CRISPR

CRISPR for Duchenne Genome editing technology to correct the mutated dystrophin gene Duchenne muscular dystrophy is caused by a mutation in the dystrophin gene, which results in the absence of full-length dystrophin protein, needed to protect muscles. Without this...

Vamorolone

Vamorolone Alternative to existing steroids for Duchenne patients The only current standard of care for boys with Duchenne is early treatment with long-term steroids, which gives some protection to muscles but with significant side effects. In July 2014, alongside...