Gene therapy involves treating illnesses by modifying, replacing, or supplementing faulty or missing genes responsible for a ...
opens new tab gene therapy for a rare bleeding disorder called ... in 60% of patients versus 29% who received infusions. Pfizer said it will continue to monitor for long-term durability and ...
the first approved gene therapy for Duchenne. Elevidys is currently only available for certain 4- to 5-year-old boys under an conditional approval granted by the FDA last June. By June 21 ...
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.
Pfizer (PFE) stock gains as lung cancer therapy, Lorbrena, shows promising results in reducing disease progression or death ...
Providing a new alternative is Tris Pharma, which has won FDA approval for Onyda XR (clonidine hydrochloride). The extended-release oral suspension becomes the first liquid non-stimulant ...
Since the FDA approved the first gene therapy in 2017, "the field is no longer in its infancy," he said. "But it's certainly not fully mature yet." Experts report drug-resistant fungi linked to ...
Pfizer (NYSE:PFE ... for both drugs was in line with previous findings. In 2021, the FDA approved Lorbrena as a first-line therapy for ALK-positive NSCLC based on CROWN data, which at the time ...
The global cell and gene therapy market is experiencing significant growth, driven by technological advancements, increasing ...
The clinical trial, which was co-led by Mass Eye and Ear in Boston and the Eye & ENT Hospital of Fudan University in Shanghai, was the first ... seeking FDA approval to bring the gene therapy ...