But the head of the FDA’s CBER office didn’t tip where the agency stands on potentially broadening use of Sarepta’s Duchenne gene therapy Elevidys.
An iEcure spokesperson said the company anticipates dosing the first patient its phase 1 study of the experimental ...
The U.S. health regulator on Wednesday approved the expanded use of Bristol Myers Squibb's cancer cell therapy Breyanzi for ...
In an important step toward more effective gene therapies for brain diseases, researchers have engineered a gene-delivery vehicle that uses a human protein to efficiently cross the blood-brain barrier ...
Hemophilia B patients given Beqvez had a decreased incidence of total bleeding Beqvez reduced the use of routine factor IX ...
Agios Pharmaceuticals announced a $905 million purchase with Royalty Pharma —Pheon Therapeutics raised a $120 million Series ...
A 12-year-old boy who suffers debilitating pain because of sickle cell disease has become the first patient in the U.S. to ...
Vertex Pharmaceuticals ( VRTX 1.36%) is worth buying today and holding forever because it has dominance in one of its core ...
Enhertu (trastuzumab deruxtecan), has become the first antibody—drug conjugate (ADC) to receive US approval for treating ...
Pfizer PFE reported the death of a young boy who participated in a mid-stage study of its Duchenne muscular dystrophy (“DMD”) ...
The drug giant is investigating with regulators and a data monitoring committee a young boy's death in a Phase II trial of fordadistrogene movaparvovec.
Alvotech and Dr. Reddy’s entered into collaboration to commercialize Prolia and Xgeva biosimilar AVT03 (denosumab) in the U.S ...