Sarepta will resume gene therapy shipments after FDA review of recent patient death
The Food and Drug Administration said it recommended lifting the hold for young patients with Duchenne's muscular dystrophy who are still able to walk. Regulators had requested the pause after the deaths of two older teenagers who were taking the therapy. The FDA also said in a statement it determined that a recently reported death of an 8-year-old boy was unrelated to the therapy.
Company shares surged more than 16% after the announcement to $13.86 in afterhours trading. The jump is the latest in a series of drastic stock movements triggered by changing fortunes for the company's best-selling product.
Elevidys is the first gene therapy approved in the U.S. for Duchenne's muscular dystrophy, the fatal muscle-wasting disease that affects boys and young men, resulting in early death. It received accelerated approval in 2023 for a narrow range of young patients and was expanded last year for use in older patients, including those who can no longer walk.
The FDA decision Monday 'significantly improves Elevidys' sales outlook in the near-term,' Jefferies analyst Andrew Tsai told investors, in a note after the announcement. 'The street will feel relieved about the situation, suggesting meaningful stock upside potential.'
Sarepta's therapy has been under scrutiny from regulators after two teenage boys died earlier this year from acute liver injury, a known side effect of the treatment. The FDA then requested a pause in shipments of the drug after the death of a third patient taking a different Sarepta therapy.
FDA officials have suggested the company will need to provide new study data on safety to resume Elevidys' use in older patients.
'The FDA will continue to work with the sponsor regarding non-ambulatory patients, which remains subject to a voluntary hold, following two deaths,' FDA said in its statement.
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The Associated Press Health and Science Department receives support from the Howard Hughes Medical Institute's Department of Science Education and the Robert Wood Johnson Foundation. The AP is solely responsible for all content.
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The design features the aforementioned 14k white gold band and a 1.5 carat princess cut lab-grown diamond. The stone is GSI-certified, ensuring the diamond is of high quality and value. Kay Jewelers The classic jewelry brand is known for more than its catchy jingle. Kay Jewelers offers a wide selection of rings to suit a variety of budgets and preferences. Plus, all of its diamonds, precious metals and gems are responsibly sourced and come backed by a lifetime guarantee. This 1.5-carat marquise lab-grown diamond sits on 14K white gold setting. Although the sparkly center stone steals most of the attention, there are smaller details that are equally as captivating. This includes the round and marquise-cut diamonds that surround the diamond across the band. Take note: the band is only available in 14k white gold at the moment, but it's ready to ship immediately. Ritani Whether you're looking for a simple design or want a more elaborate look to your engagement ring, Ritani has you covered. Right now, you can customize your diamond or lab-grown diamond ring for as low as $1,600. The retailer has countless affordable rings, but you can increase your savings by joining Ritani's email list to secure $100 off your order. For a limited time, take up to 40% off select loose lab-grown diamonds with code FLASH40. Or, get up to 20% off lab-grown diamonds for your customizable engagement ring with code LAB20. The 'Toi et moi' or 'you and me' engagement ring style is the perfect way to symbolize your upcoming marriage due to its unique two-stone design. Personalize the ring by selecting the second stones shape, metal and carat size. The current offer above includes a .50-carat pear diamond. Depending on your preferences, the price of the ring may increase. Vrai Our editors named Vrai the best place to purchase lab-grown diamond engagement rings thanks to its sustainable practices, high-quality stones and sophisticated designs. The retailer offers made-to-order and ready-to-ship engagement rings as low as $1,900. This simple ring has a 1.65-carat lab-grown center diamond secured by a 14k yellow gold band. A hidden halo holds even more handset pavé diamonds, so it shines from all angles. To maintain the look and feel of the ring, it also comes with Vrai's care package, which includes warranty, annual shine service and complimentary resizing. Grown Brilliance Our editors love Grown Brilliance because of its Truly Custom program—you send in inspiration images, and a jeweler will create a one-of-a-kind piece for you. But it also offers plenty of high-quality, pre-designed engagement rings that are on the affordable end of the spectrum. Emerald cuts rose in popularity during the Art Deco era, and they appear to be making a comeback. This stunning, vintage-inspired design combines intricate details and shapes to create a cohesive work of art. The ring features a lab-grown emerald diamond at its center, accompanied by additional stones that build upon it, as well as the band itself. Although you can't customize its shape, you can choose from white gold, yellow gold, rose gold and platinum bands. Quince Our team named Quince the best retailer for affordable engagement rings. 'Its pieces are stunning and well made,' says deputy editor Kari Molvar, who is a certified gem expert. The site offers both high-quality lab-grown and natural diamond rings—without the luxury price point. At Quince, find timeless and classic stones (including the setting) for as low as $900. This carefully sourced and crafted engagement ring is a true showstopper. At its center sits a round lab-grown diamond, surrounded by a halo of smaller stones that amplify its shine from every angle. The ring is available in 15 sizes, three carat weights and three metal options. With Clarity Another editor- and expert-approved engagement ring site is With Clarity, a retailer that allows you to build your ring or choose from its ready-made natural or lab-grown styles. Right now, you can shop rings for as low as $1,200 and also get up to 25% off select styles. For those who want a slim band with subtle accents, this With Clarity engagement ring is an excellent choice. The ring shown above features 14K yellow gold and a 2-carat lab-grown diamond with tiny, round stones set alongside it. However, if you prefer a different carat weight, diamond shape or metal, you can customize it to your liking. You can also add a meaningful touch to the band by personalizing it with 15 characters. Jared Jewelers This tried-and-true jewelry brand is renowned for offering a diverse selection of pieces suitable for all occasions, including engagements. Get up to 70% off select clearance styles and 20% off storewide. You can shop for premade options, customizable rings or designer rings from Vera Wang VOW, Chosen, J'Lure and more. Although Jared Jewelers has countless options, this classic, ready-to-ship design is available for under $1,100. Reviewers say that this simple ring is a perfect option because it's stunning without being overly expensive. Macy's The popular retailer sells a variety of jewelry pieces, including hundreds of engagement rings from brands like Badgley Mischka and Suzy Levian New York, with plenty of options priced below $5,000. You can also maximize your savings by taking advantage of the best Macy's promo codes right now. This modern engagement ring features round Moissanite stones and a knife-edge design—a raised ridge that runs along the band. After purchase, it can be registered under Macy's WorryNoMore Jewelry and Watch Protection program, which covers future services such as resizing, resetting and more. The ring is also covered by Charles & Colvard's lifetime warranty, a benefit that protects optical properties and physical integrity. Angara Angara is an online jewelry brand that offers a wide selection of cuts, gemstones and metals to help you discover your ideal engagement ring. Browse hundreds of options to find your personal style, including vintage-inspired designs and three-stone settings. For a limited time, you can also score up 10% off sitewide. This modern take on a classic style showcases a pear-shaped diamond placed in a bezel setting. The ring pictured above is a 2-carat lab-grown diamond with a 14K gold band and F-G quality. If you wish to make alterations, the ring is available in five gemstone qualities, four colors, 23 sizes and comes with free personalized engraving. Rare Carat Rare Carat offers a wide variety of rings (over 1,000 settings and diamonds), making it possible to find the perfect option for your soon-to-be bride. 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Bliss Diamond & Jewelry Co. is a family-owned business that offers high-quality, lab-grown diamonds and jewelry, including this 3-carat engagement ring. This dazzling cushion-cut diamond ring, which is available in 10 sizes, can be set in 14K white gold or 14K yellow gold. What Is A Reasonable Engagement Ring Price? A reasonable engagement ring price largely depends on your budget. You should factor in the cost of the ring itself, taxes, insurance and any personalization details. However, a quality diamond paired with its setting typically starts at around $2,000 and increases from there. Still, there are plenty of beautiful rings available that are less expensive than the average ring price. What Is The Most Affordable Engagement Ring Site? There are a handful of engagement ring websites that offer options to meet most budgeting needs and style preferences. After assessing multiple factors, our team named Quince as the site with the best affordable engagement rings. Quince offers high-quality rings at a low cost, including both lab-grown and natural diamonds, and has a generous return window. How Can You Shop For Affordable Engagement Rings On A Budget? When shopping for an affordable engagement ring, it's essential to set a budget for yourself before you start looking. To maximize your spending, consider purchasing a lab-grown diamond, as these tend to be more cost-effective than mined diamonds. Characteristics and qualities to consider when looking at natural diamonds include the Four C's (cut, color, clarity and carat weight), certifications, metals and diamond setting. What Is The Cheapest Month To Buy An Engagement Ring? You can find discounts on engagement rings throughout the year, but certain months typically offer better deals than others. For example, jewelry sales tend to slow down at the end of summer, making it a good time to score savings during this quieter shopping period. The holiday season—particularly November and December—often brings a wave of promotions to attract gift-givers. Following the holidays, January is also considered a slow month for jewelry retailers, so you may see additional sales aimed at boosting post-holiday purchases.
Wall Street Journal
a minute ago
- Wall Street Journal
Real Strains Inside the BLS Made It Vulnerable to Trump's Accusations
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