A bellwether moment for a gene therapy; Uber's pilot of free rides to prenatal appointments

For Sarepta, an experimental gene therapy is a story of foresight, savvy dealmaking, scientific puzzle solving, and perseverance.

April 17, 2023 Check out STAT+

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Maddie McGarvey for STAT STAT+ | A bellwether moment: Once a distant dream, gene therapy for Duchenne nears historic decision A looming verdict on Sarepta's therapy has made this perhaps the most consequential moment in the history of Duchenne. By Jason Mast and Adam Feuerstein STAT+ | Why do some blood stem cells go rogue? Study offers clue and possible target Many people have blood cell clones that have the potential to turn cancerous. A specific DNA change is now identified as a key. By Angus Chen Free Uber rides helped patients keep their prenatal appointments. Now the company wants insurers to pay for it Using a consumer app like Uber could make the process of organizing and taking rides to a clinic easier both for staff and patients. By Mohana Ravindranath KENA BETANCUR/AFP via Getty Images Opinion: OTC naloxone is a baby step toward making the life-saving medication accessible Now that naloxone will be available OTC, we need more substantial efforts to reduce cost and increase access to this lifesaving medication. By Jonathan JK Stoltman and Mishka Terplan Q&A: What does it mean when a clinical trial is delayed? More than you think STAT sat down with clinical trials expert Frank David to talk about why trial delays are important and how trials have changed over time. By Erica Goode Opinion: Where are the guidelines for the production of animals with intentional genomic alterations? The public should not have to settle for reading regulatory tea leaves when it comes to animals with intentional genomic alterations. By Peter G. Lurie and Beth Ellikidis

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