An 8-year-old northern Minnesota boy is one of the first in the country to receive a specific type of gene therapy treatment.

Study results support the role of functional dystrophin and suggest that delandistrogene moxeparvovec stabilizes or slows ...

About three months after leaving her post as the FDA’s principal deputy commissioner, Namandjé Bumpus, Ph.D., has found a new ...

Preclinical data with the investigational gene therapy INS1202 show improvements in survival and motor performance in ALS.

Data from the SAPPHIRE study formed the basis of Scholar Rock's U.S. Food and Drug Administration request to approve apitegromab for SMA.