Spinal muscular atrophy with respiratory distress type 1 (SMARD1) and type 2 (SMARD2) are caused by mutations in different genes. Both conditions are rare, but researchers know less about SMARD2.

The news: Myosana Therapeutics, a Seattle startup developing gene therapy technology, raised $5 million to develop an early-stage candidate treatment for Duchenne muscular dystrophy (DMD).

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 ...