Por: The Boston Globe Business May 13, 2023
An panel of advisers Friday recommended that the Food and Drug Administration speed up approving a Duchenne muscular dystrophy treatment developed by Cambridge biotech Sarepta Therapeutics, despite questions about its clinical benefit and safety.By an 8 to 6 vote, the FDA advisers endorsed accelerated approval for the experimental gene therapy, called SRP-9001. The vote followed a daylong hearing, convened virtually, marked by sharp debate over... + full article
The Boston Globe USA Business May 24, 2023
The Food and Drug Administration is delaying by one month its long-awaited decision on whether to approve a gene therapy for a deadly inherited muscle-wasting disease that afflicts more than 10,000 boys and young men nationwide, according to the Cambridge biotech that developed... + más
FDA advisers recommend accelerated approval of a Sarepta gene therapy | The Boston Globe
First gene therapy for deadly form of muscular dystrophy gets FDA approval for young kids | ABC News
RTTNews USA Health May 24, 2023
Sarepta Therapeutics, Inc. () on Wednesday provided the following update on the Biologics License Application (BLA) for SRP-9001 (delandistrogene moxeparvovec), which is currently under review for the treatment of ambulant individuals with Duchenne muscular dystrophy (DMD) who... + más
Sarepta Submits BLA For SRP-9001 To Treat Ambulant Patients With Duchenne Muscular Dystrophy | RTTNews
The Boston Globe USA Business May 23, 2023
Regulators are to decide within days whether to clear a Cambridge biotech’s gene therapy for a deadly muscle-wasting disease that afflicts more than 10,000 boys and young men nationwide, even though questions remain about whether the treatment is safe and effective.The... + más
Gene therapy has made some recent progress—is it enough? | Ars Technica
Ars Technica USA Science January 05, 2023
Navigate Filter by topic Settings Front page layout Site theme - Jan 5, 2023 12:45 pm UTC MARK GARLICK/SCIENCE PHOTO LIBRARY Share this story Gene therapy has had a long and bumpy history. Although researchers have made some notable and Despite these issues, experts say there... + más
Opinion | The New York Times
First participant in test of custom gene editing therapy dies | The Boston Globe
The New York Times USA Opinion December 09, 2022
The parents of a 2-year-old girl write that their daughter “could die within the next year” because a genetic mutation is causing her heart to fail.“Time is quickly running out for me,” writes a man in his mid-30s whose DNA harbors a genetic mistake certain to destroy... + más
Justice Alito accused of being SCOTUS leaker after new report: Disgrace | Newsweek
Why would Mac Jones get a second opinion on his injury, and how common is that? | The Boston Globe
CBS News USA Health November 23, 2022
Washington — U.S. health regulators on Tuesday approved the first gene therapy for hemophilia, a $3.5 million one-time treatment for the blood-clotting disorder. The Food and Drug Administration cleared Hemgenix, an IV treatment for adults with hemophilia B, the less common... + más
$3.5M gene therapy for hemophilia gets FDA approval | ABC News
$3.5M gene therapy for hemophilia gets FDA approval | WPLG Local 10
RTTNews USA Health September 29, 2022
Genetic medicine maker Sarepta Therapeutics, Inc. () announced Thursday that it has submitted a Biologics License Application (BLA) to the U.S. Food and Drug Administration (FDA) for the accelerated approval of SRP-9001 (delandistrogene moxeparvovec) to treat ambulant patients... + más
Diageo Debuts Its 2022 Special Releases Collection | Forbes
Parents of son diagnosed with Duchenne muscular dystrophy are hopeful cure will be found soon | ABC7
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