gene therapy

Gene therapy can cure rare diseases. But drug makers have little incentive, leaving families desperate

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Robin Alderman faces a painful reality: gene therapy could cure her son Camden’s rare hereditary immune deficiency. But it’s not for him. In 2022, London-based Orchard Therapeutics stopped investing in an experimental treatment for the condition, Wiskott-Aldrich syndrome. And there are no gene therapy studies he can participate in. “We feel like we’ve been forgotten,” … Read more

Gene therapy can cure rare diseases. But drug makers have little incentive, leaving families desperate

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Robin Alderman faces a painful reality: gene therapy could cure her son Camden’s rare hereditary immune deficiency. But it’s not for him. In 2022, London-based Orchard Therapeutics stopped investing in an experimental treatment for the condition, Wiskott-Aldrich syndrome. And there are no gene therapy studies he can participate in. “We feel like we’ve been forgotten,” … Read more

Girl born deaf is the first in the world to have her hearing restored in a new gene therapy study

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A British girl has had her hearing restored after becoming the first person in the world to take part in a groundbreaking new gene therapy trial. Opal Sandy, 18 months old, was born completely deaf due to the condition auditory neuropathy, which is caused by the disruption of nerve impulses traveling from the inner ear … Read more

Toddler born deaf is the first in the world to have his hearing restored in a new gene therapy study

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A British girl has had her hearing restored after becoming the first person in the world to take part in a groundbreaking new gene therapy trial. Opal Sandy, 18 months old, was born completely deaf due to the condition auditory neuropathy, which is caused by the disruption of nerve impulses traveling from the inner ear … Read more

British girl has her hearing restored as the first patient in a new gene therapy trial

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A British girl has had her hearing restored after becoming the first person in the world to take part in a groundbreaking new gene therapy trial. Opal Sandy, 18 months old, was born completely deaf due to the condition auditory neuropathy, which is caused by the disruption of nerve impulses traveling from the inner ear … Read more

Couple’s joy when deaf daughter hears thanks to new gene therapy

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The parents of an 18-month-old girl who can hear thanks to a groundbreaking gene therapy for deafness have said they are pleased with her progress. Jo Sandy, a 33-year-old secondary school geography teacher, and her husband James, 33, who works in car manufacturing, said they were “stunned” when they realized Opal could hear without the … Read more

Thanks to gene therapy, an 11-year-old boy can hear for the first time

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Dr. John Germiller, an otolaryngologist who leads the CHOP study, at the Children’s Hospital of Philadelphia on Jan. 16, 2024. (Hannah Beier/The New York Times) PHILADELPHIA — Aissam Dam, an 11-year-old boy, grew up in a world of deep silence. He was born deaf and had never heard anything. While living in a poor community … Read more

Thanks to gene therapy, an 11-year-old boy can hear for the first time

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PHILADELPHIA — Aissam Dam, an 11-year-old boy, grew up in a world of deep silence. He was born deaf and had never heard anything. While living in a poor community in Morocco, he expressed himself using a sign language he invented and had no education. Last year, after he moved to Spain, his family took … Read more