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Engels

US approves first gene therapy for rare form of hearing loss

WASHINGTON - US health officials on Thursday greenlit a first-of-its-kind gene therapy to treat a rare form of hereditary hearing loss,...

Times of Suriname

a breakthrough which could pave the way for other such hearing impairment treatments. Two to three of every 1,000 children in the United States are born with a hearing impairment, and it is estimated that more than half of these cases of early-onset hearing loss are caused by genetic mutations. Developed by the American biotechnology company Regeneron, the treatment known as Otarmeni targets a rare form of hearing loss which affects around 50 newborns a year in the US. It will be available for children and adults with severe-to-profound hearing loss caused by certain mutations in the OTOF gene, which encodes a protein critical for transmitting auditory signals from the inner ear to the brain.

While gene therapies are generally extremely expensive -- particularly in the United States, where they can cost several million dollars per patient -- Regeneron said it intends to offer this treatment free of charge to eligible American patients. Administered as a single injection into the ear by a surgeon, the treatment has been hailed as revolutionary by parents of affected children.  "It's absolutely incredible," said an emotional Sierra Smith, the young mother of Travis, a baby boy who received the treatment.

The clinical trial, in which 20 pediatric patients aged 10 months to 16 years were evaluated, at least 80 percent experienced a significant improvement in hearing after a few months. The US Food and Drug Administration's (FDA) accelerated approval of this treatment "signals a new era in the treatment of genetic forms of hearing loss, where reinstating 24/7 natural hearing is now possible," said Eliot Shearer, a doctor involved in the trial. (Bssnews)

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