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For Ashton Drake GENE Doll OAK 1997 “For the Boys”
Tag: gene
FDA approves Pfizer’s first gene therapy for rare inherited bleeding disorder
The Food and Drug Administration on Friday approved Pfizer’s treatment for a rare genetic bleeding disorder, making it the company’s first-ever gene therapy to win clearance in the U.S. The agency greenlit the drug, which will be marketed as Beqvez, for adults with moderate to severe hemophilia B who meet certain requirements. The treatment will […]
Gene mutation that ‘reduces odds of Alzheimer’s disease by 70%’ discovered
A gene mutation that reduces the odds of developing Alzheimer’s disease by 70 per cent has been discovered by scientists. This “protective” variant occurs in a gene – FN1 – which makes a protein known as fibronectin. The discovery was made in people who had another gene known as APOEe4, which significantly increases the risk […]
Experimental gene therapy allows kids with inherited deafness to hear
Gene therapy has allowed several children born with inherited deafness to hear. A small study published Wednesday documents significantly restored hearing in five of six kids treated in China. On Tuesday, the Children’s Hospital of Philadelphia announced similar improvements in an 11-year-old boy treated there. And earlier this month, Chinese researchers published a study showing […]
Gene Therapy Allows an 11-Year-Old Boy to Hear for the First Time
PHILADELPHIA — Aissam Dam, an 11-year-old boy, grew up in a world of profound silence. He was born deaf and had never heard anything. While living in a poor community in Morocco, he expressed himself with a sign language he invented and had no schooling. Last year, after moving to Spain, his family took him […]
VYGR Stock Catapults Higher After Novartis Sweetens Its Gene Therapy Deal
Novartis (NVS) expanded its gene therapy collaboration with Voyager Therapeutics (VYGR) on Monday. VYGR stock rocketed above its 200-day line for the first time in four months.
Novartis signs gene therapy deal with Voyager for $100 million upfront
(Reuters) -Voyager Therapeutics said on Tuesday Swiss-based drugmaker Novartis would pay $100 million upfront as part of a licensing deal to develop gene therapy candidates for genetic disorders. Voyager, whose shares jumped more than 30% in premarket trading, would be eligible to receive up to $1.2 billion on achieving certain milestones. The gene therapy developer […]
Sickle cell affects more families in Africa and India, but new gene therapies are out of reach
Gautam Dongre’s two children in India and Pascazia Mazeze’s son in Tanzania live with an inherited blood disorder that turns blood cells into instruments of pain. Now that new gene therapies promise a cure for their sickle cell disease, Dongre says he’s “praying the treatment should come to us.” But experts say the one-time treatment […]
US regulators approve two gene therapies for sickle cell disease | US news
The US Food and Drug Administration has approved a pair of gene therapies for sickle cell disease, including the first treatment based on the breakthrough Crispr gene-editing technology, opening up two “transformative therapy” avenues for some patients. The FDA approved Lyfgenia from Bluebird Bio, and a separate treatment called Casgevy by partners Vertex Pharmaceuticals and […]