Last week, an FDA advisory committee voted in favor of approving Sarepta Therapeutics, Inc.'s SRP-9001 for treating DMD. Sarepta Therapeutics stock has good near-term momentum as the BLA is scheduled ...
In the avian embryo, vascular smooth muscle cells (VSMC) in the aortic arch (elastic) arteries originate in the neural crest, whereas other VSMC develop from local mesoderm. These two lineages have ...
The investigational therapy uses an adeno-associated viral (AAV) vector (serotype: AAV MYO2) encoding the fukutin-related protein (FKRP) under the control of a muscle-specific promoter to restore ...