Pfizer (PFE) stock gains as lung cancer therapy, Lorbrena, shows promising results in reducing disease progression or death ...
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.
The European Medicines Agency has recommended the use of Pfizer's gene therapy for a rare bleeding disorder called hemophilia ...
Researchers are engineering ways to alter genes or swap flawed ones with healthy replacements, hoping to cure diseases or enable the body to better combat them.
Ultragenyx is gearing up to take its glycogen storage disease (GSD) treatment to regulators next year after the gene therapy ...
Kendric Cromer is the first commercial patient to receive the groundbreaking treatment. Kendric Cromer, 12, is the first patient in the United States to undergo a newly approved gene therapy for ...
Stimulants are typically the go-to option for doctors prescribing treatments for attention-deficit hyperactivity disorder (ADHD). But some ADHD patients do not respond adequately to stimulants or ...
When we think of smoking-related cancers, lung cancer usually comes to mind. But smoking is also a major driver for bladder cancer. Now, a new treatment has come to Philadelphia to help more ...
The global cell and gene therapy market is experiencing significant growth, driven by technological advancements, increasing ...
Eladocagene exuparvovec is a one-time, recombinant adeno-associated virus serotype 2-based gene replacement therapy.
Eli Lilly’s Retevmo (selpercatinib) has won an FDA accelerated approval to treat certain kids ages two and older with either advanced or metastatic medullary thyroid cancer (MTC) with a RET ...
The drug is a potential treatment for biliary atresia in patients who have undergone surgery in which blocked bile ducts are bypassed to restore normal bile flow. The growth was driven by spending on ...