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Stockreport

FDA approves first gene therapy for inherited deafness, shown to restore hearing for children with rare condition [CNN]

Regeneron Pharmaceuticals, Inc. (REGN)  More Company Research Source: CNN
Last regeneron pharmaceuticals, inc. earnings: 2/6 06:30 am Check Earnings Report
US:NASDAQ Investor Relations: investor.regeneron.com
The condition it treats is rare, affecting up to about 50 babies born each year in the US with mutations in a gene known as OTOF. But the therapy's effect can be profound: In a clinical trial of 20 kids, 16 had improvements in hearing about five months after treatment. Five of 12 that were followed for at least 11 months had their hearing essentially restored to normal. “It's miraculous,” Kerri, the mother of a clinical trial participant who requested that her last name be withheld, told CNN. Her 2-year-old son, Miles, had the gene therapy delivered to both ears in May. “You go from being told your child's profoundly deaf and may only ever hear with technology to your child's hearing right alongside his friends. … This is just amazing.” The gene therapy is made by biotechnology company Regeneron, which also made news with its planned price for the medicine. Often, one-time gene therapies for rare conditions are priced by manufacturers at millions of dollars per patient in order t [Read more]
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Stockreport

FDA approves first gene therapy for inherited deafness, shown to restore hearing for children with rare condition [CNN]

Regeneron Pharmaceuticals, Inc.  (REGN) 
More Company Research Source: CNN
Last regeneron pharmaceuticals, inc. earnings: 2/6 06:30 am Check Earnings Report
US:NASDAQ Investor Relations: investor.regeneron.com
The condition it treats is rare, affecting up to about 50 babies born each year in the US with mutations in a gene known as OTOF. But the therapy's effect can be profound: In a clinical trial of 20 kids, 16 had improvements in hearing about five months after treatment. Five of 12 that were followed for at least 11 months had their hearing essentially restored to normal. “It's miraculous,” Kerri, the mother of a clinical trial participant who requested that her last name be withheld, told CNN. Her 2-year-old son, Miles, had the gene therapy delivered to both ears in May. “You go from being told your child's profoundly deaf and may only ever hear with technology to your child's hearing right alongside his friends. … This is just amazing.” The gene therapy is made by biotechnology company Regeneron, which also made news with its planned price for the medicine. Often, one-time gene therapies for rare conditions are priced by manufacturers at millions of dollars per patient in order t [Read more]
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