India, July 23 -- Avidity Biosciences Inc.'s (RNA) investigational ribonucleic acid therapy Del-zota has received Breakthrough Therapy designation from the FDA for the treatment of Duchenne muscular dystrophy in people living with mutations amenable to exon 44 skipping.
Breakthrough Therapy designation is a process designed to expedite the development and review of drugs that are intended to treat a serious condition and preliminary clinical evidence indicates that the drug may demonstrate substantial improvement over available therapy on a clinically significant endpoint(s).
Duchenne muscular dystrophy (DMD) is a rare genetic condition that is characterized by progressive muscle damage and weakness due to the loss of dystrophin protein, ...