Gene therapy

Dr. Jason Comander, inherited retinal disorder specialist at Massachusetts Eye and Ear Infirmary in Boston points to a model of an eye during an interview on Jan. 8, 2020. Comander's hospital plans to enroll patients in a gene editing treatment for blindness study. He said it marks “a new era in medicine” using a technology that “makes editing DNA much easier and much more effective.” (AP Photo/Rodrique Ngowi)
March 04, 2020 - 5:03 am
Scientists say they have used the gene editing tool CRISPR inside someone's body for the first time, a new frontier for efforts to operate on DNA, the chemical code of life, to treat diseases. A patient recently had it done at the Casey Eye Institute at Oregon Health & Science University in...
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FILE - This May 26, 2009 file photo shows a printout from an electrocardiogram machine in Missouri. Doctors are reporting that novel drugs may offer fresh ways to reduce heart risks beyond the usual medicines to lower cholesterol and blood pressure. One new study found that heart attack survivors benefited from a medicine long used to treat gout. Gene-targeting medicines also showed promise in studies discussed Monday, Nov. 18, 2019, at an American Heart Association conference in Philadelphia. (AP Photo/Jeff Roberson)
November 18, 2019 - 3:26 pm
PHILADELPHIA (AP) — Novel drugs may offer fresh ways to reduce heart risks beyond the usual medicines to lower cholesterol and blood pressure. One new study found that heart attack survivors benefited from a medicine long used to treat gout. Several experimental drugs also showed early promise for...
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FILE - This May 26, 2009 file photo shows a printout from an electrocardiogram machine in Missouri. Doctors are reporting that novel drugs may offer fresh ways to reduce heart risks beyond the usual medicines to lower cholesterol and blood pressure. One new study found that heart attack survivors benefited from a medicine long used to treat gout. Gene-targeting medicines also showed promise in studies discussed Monday, Nov. 18, 2019, at an American Heart Association conference in Philadelphia. (AP Photo/Jeff Roberson)
November 18, 2019 - 2:12 pm
PHILADELPHIA (AP) — Novel drugs may offer fresh ways to reduce heart risks beyond the usual medicines to lower cholesterol and blood pressure. One new study found that heart attack survivors benefited from a medicine long used to treat gout. Several experimental drugs also showed early promise for...
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FILE - In this Feb. 14, 2019, file photo, Centers for Medicare & Medicaid Services (CMS) Administrator Seema Verma speaks during a news conference in Washington. Expanding access to a promising but costly treatment, Medicare said Aug. 7, it will cover for some blood cancers a breakthrough gene therapy that revs up a patient’s own immune cells to destroy malignancies. Verma said the decision will provide consistent and predictable access nationwide, opening up treatment options for some patients "who had nowhere else to turn." (AP Photo/Kevin Wolf, File)
August 07, 2019 - 6:44 pm
WASHINGTON (AP) — Expanding access to a promising but costly treatment, Medicare said Wednesday it will cover for some blood cancers a breakthrough gene therapy that revs up a patient's own immune cells to destroy malignancies. Officials said Medicare will cover CAR-T cell therapies for certain...
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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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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 - 6:24 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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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 - 2:35 pm
U.S. regulators have approved the most expensive medicine ever, for a disorder that destroys a baby's muscle control and kills nearly all of those with the most common variant of the disease within a couple years. The treatment is priced at $2.125 million. Out-of-pocket costs for patients will vary...
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May 24, 2019 - 1:41 pm
U.S. regulators have approved the most expensive medicine ever, a therapy meant to cure a disorder that rapidly destroys a baby's muscle control and kills most within a couple years. The one-time gene therapy developed by Novartis, Zolgensma (zohl-JEN-smah), will cost $2.125 million. It treats a...
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