Gene therapies and research into them have grown immensely in recent years, offering more novel tools in regenerative medicine to fight disease, including rare diseases and genetic disorders.
The FDA has accelerated its review of Novartis’ sickle cell drug crizanlizumab after the company filed phase 2 results showing its effectiveness as a treatment for life threatening and pain
The FDA has granted Breakthrough Therapy designation to Novartis' crizanlizumab for the prevention of vaso-occlusive crises (VOCs) in patients of all genotypes with