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FDA approves first gene therapy for genetic hearing loss [MSNBC.com]
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US:NASDAQ Investor Relations: investor.regeneron.com
The Food and Drug Administration on Thursdayapprovedgene therapyThe drugmaker said it will offer the drug for free to U.S. patients.Otarmeni is approved to treat a very rare form of hearing loss that affects about 50 babies born in the U.S. each year and is caused by a mutation in a gene called OTOF. The approval was granted under theCommissioner’s National Priority Voucher“This really is life-changing for families with children with hearing loss,” Dr. Eliot Shearer, a pediatric otolaryngologist at Children’s Hospital and a principal investigator on the Regeneron trial.The only other treatment option for children with genetic deafness is cochlear implants, Shearer said, which restore the ability to hear speech and music, but reduce the fidelity of the sound.With gene therapy, the improved hearing is “on 24/7 and doesn’t rely on batteries,” he added.The drug works by replacing the faulty OTOF gene, which is supposed to work by giving the body instructions to make a protein called oto
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- Otarmeni™ (lunsotogene parvec-cwha) Approved by FDA as First and Only Gene Therapy for Genetic Hearing Loss; Regeneron to Provide Otarmeni for Free in the U.S. [Yahoo! Finance][Yahoo! Finance]
- 'Two ways of calculating': Trump defends his mathematically impossible calculations on drug prices [Yahoo! Finance][Yahoo! Finance]
- Regeneron Announces Agreement with U.S. Government to Help Lower Drug Costs for American Patients and Will Provide Innovative New Gene Therapy for Free in the U.S.[GlobeNewswire]
- Regeneron inks drug pricing deal with Trump, will offer new hearing-loss therapy for free [CNBC][CNBC]
- FDA approves first gene therapy for inherited deafness, shown to restore hearing for children with rare condition [CNN][CNN]
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