Gene therapy

This photo provided by Novartis shows their gene therapy medicine Zolgensma. U.S. regulators want to know why Novartis didn't disclose a problem with testing data until after they approved the Swiss drugmaker’s $2.125 million gene therapy. On Tuesday, Aug. 6, 2019, the Food and Drug Administration said the questionable data involves testing of the therapy, Zologensma, on animals, not on patients. (Novartis via AP)
August 06, 2019 - 6:51 pm
TRENTON, N.J. (AP) — U.S. regulators want to know why Novartis didn't disclose a problem with testing data until after the Swiss drugmaker's $2.1 million gene therapy was approved. The Food and Drug Administration said Tuesday the manipulated data involved testing in animals, not patients, and it's...
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In this June 20, 2019 photo, the Capitol is seen from the roof of the Canadian Embassy in Washington. Health care is on the agenda for Congress when lawmakers return, and it’s not another battle over the Obama-era Affordable Care Act. Instead of dealing with the uninsured, lawmakers are trying to bring down costs for people who already have coverage. (AP Photo/J. Scott Applewhite)
July 07, 2019 - 9:00 am
WASHINGTON (AP) — Lawmakers are trying to set aside their irreconcilable differences over the Obama-era Affordable Care Act and work to reach bipartisan agreement on a more immediate health care issue, lowering costs for people who already have coverage. Returning from their Fourth of July recess,...
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This photo provided by Novartis shows Zolgensma. The one-time gene therapy developed by Novartis, Zolgensma, will cost $2.125 million. It treats a rare condition called spinal muscular atrophy, or SMA, which strikes about 400 babies born in the U.S. each year. The therapy, given in a one-hour infusion, was approved for children under age 2 and will be available within two weeks. (Novartis via AP)
May 24, 2019 - 5:51 pm
U.S. regulators have approved the most expensive medicine ever, for a rare disorder that destroys a baby's muscle control and kills nearly all of those with the most common type of the disease within a couple of years. The treatment is priced at $2.125 million. Out-of-pocket costs for patients will...
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February 25, 2019 - 2:40 pm
Stocks marched higher on Wall Street Monday after President Donald Trump agreed to hold off on raising tariffs on Chinese goods, which would have escalated a damaging trade war between the world's two largest economies. Investors had been growing increasingly optimistic over the last two weeks that...
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FILE - In this Monday, Nov. 13, 2017 file photo, Brian Madeux, starts to receive the first human gene editing therapy for Hunter syndrome, as his girlfriend, Marcie Humphrey, left, applauds at the UCSF Benioff Children's Hospital in Oakland, Calif. At right is nurse practitioner Jacqueline Madden. On Thursday, Feb. 7, 2019, scientists gave an update on the first effort to edit genes, or permanently change the DNA, of about a dozen adults, including Madeux, with metabolic diseases. (AP Photo/Eric Risberg)
February 07, 2019 - 12:05 pm
Scientists think they have achieved the first gene editing inside the body, altering DNA in adults to try to treat a disease, although it's too soon to know if this will help. Preliminary results suggest that two men with a rare disorder now have a corrective gene at very low levels, which may not...
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In this Monday, Aug. 13, 2018 photo, Brian Madeux interacts with research nurse Chrishauna Lacy at his home in New River, Ariz. Madeux was the first person in the world to participate in a gene editing attempt in his body, for the inherited disease Hunter syndrome. Early partial results from the study were released on Wednesday, Sept. 5, 2018. (AP Photo/Matt York)
September 05, 2018 - 8:58 am
PHOENIX (AP) — Early, partial results from a historic gene editing study give encouraging signs that the treatment may be safe and having at least some of its hoped-for effect, but it's too soon to know whether it ultimately will succeed. The results announced Wednesday are from the first human...
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FILE - In this Monday, Nov. 13, 2017 file photo, a nurse practitioner prepares to start the first human gene editing treatment for Hunter syndrome, an inherited metabolic disease, at a hospital in Oakland, Calif. On Wednesday, Aug. 15, 2018, federal officials said that gene therapy is becoming an established form of medical care and carries no special risks that warrant special regulation, as they revised rules for vetting such experiments and products. (AP Photo/Eric Risberg)
August 15, 2018 - 6:57 pm
U.S. health officials are eliminating special regulations for gene therapy experiments, saying that what was once exotic science is quickly becoming an established form of medical care with no extraordinary risks. A special National Institutes of Health oversight panel will no longer review all...
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