Spinal muscular atrophy with respiratory distress (SMARD) is a rare genetic condition that typically affects infants and children. It causes muscle weakness and breathing problems. Spinal muscular ...

From left: Myosana CEO Matthew Lumley, with co-founders Stanley Froehner and Nick Whitehead. (Myosana and UW Photos) The news: Myosana Therapeutics, a Seattle startup developing gene therapy ...

Duchenne muscular dystrophy (DMD) is a muscle degeneration disorder caused by mutations affecting the dystrophin gene. Researchers show how a dual CRISPR RNA method restored dystrophin protein ...