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FDA asks Sarepta to stop shipping gene therapy
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Groundbreaking new research shows that it is possible to use gene therapy to restore hearing in both children and adults.
The Centers for Medicare and Medicaid Services (CMS) has selected 35 participants for its new, voluntary Cell and Gene Therapy Access Model. | As expected, there will be 35 voluntary participants in the CMS' new Cell and Gene Therapy Access Model.
Since gene therapy was first proposed to treat such genetic diseases in the 1970s, it has had thrilling highs — including the first successful gene therapy, in 1990, in a 4-year-old born with ...
Gene therapy can treat certain blood diseases, such as hemophilia A, hemophilia B, sickle cell disease, and as of 2022, beta thalassemia.
A team of Chinese medical experts say they can produce anti-cancer cells directly inside the human body using gene therapy tools.
Massachusetts-based Sarepta Therapeutics, facing scrutiny from regulators, is reducing its global workforce by 36% in an effort to cut annual operating costs by hundreds of millions of dollars.
Shares of Sarepta Therapeutics plunged more than 30% on Friday as the future of its approved gene therapy appeared at risk. Sarepta has reported three patient deaths related to its gene therapies.
Our Bureau, Mumbai Friday, July 18, 2025, 17:45 Hrs [IST]
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