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☆ Ohio woman charged after police say she faked her daughter's cancer diagnosis to raise thousands of dollars
▼ + stars: | 2024-01-13 | by ( Kenneth Niemeyer | ) www.businessinsider.com time to read: +3 min

Ohio authorities have accused a woman of faking her daughter's cancer diagnosis to raise money. download the app Email address Sign up By clicking “Sign Up”, you accept our Terms of Service and Privacy Policy . AdvertisementAuthorities in Ohio say a woman faked her daughter's cancer diagnosis and used it to raise thousands of dollars in online fundraisers for her treatment. The sheriff's office said it received a tip that a local child was being used to improperly raise money based on the public portrayal that they had cancer. AdvertisementAuthorities said in the document that school officials noted that Reed's child has already missed more than 280 hours of school this school year.

Persons: Pamela Reed, —, Reed, isn't, Kelly Turner, Olivia, Turner, Gypsy Rose Blanchard, Dee Dee, Blanchard, Rose Blanchard, Nicholas Godejohn, Clauddine Blanchard, Godejohn Organizations: Service, Sheriff's, Law &, Reed's, Authorities, The New York Times, Times, Humanity Locations: Ohio, Noble, United States, Colorado, Habitat

☆ Fresh from a murder sentence, Gypsy Rose Blanchard is becoming the internet's favorite influencer
▼ + stars: | 2024-01-10 | by ( Lindsay Dodgson | ) www.businessinsider.com time to read: +9 min

The 32-year-old was recently released from prison after eight years for orchestrating the murder of her mother, Clauddine "Dee Dee" Blanchard. Where Gypsy Rose Blanchard's story beganDee Dee Blanchard and Gypsy Rose Blanchard. AdvertisementBehind closed doors Dee Dee was overbearing, but to the outside world, it was a different story. She was also a lot older than Dee Dee was telling people she was. In one with People after her release, she said killing Dee Dee was an act of "panic" and "desperation" because she was facing yet another surgery.

Persons: —, Rose Blanchard, Dee Dee, Blanchard, Blanchard shouldn't, Elizabeth Holmes, Nina Vargas, Dee Dee Blanchard, Gypsy Rose Blanchard, Joey King, Patricia Arquette, Dee Dee's, Nicholas Godejohn, Dee, Godejohn, I'm, Blanchard hasn't, She's, Claudia Oshry, Time's, Amanda Hirsch, notskinnybutnotfat, Ryan Anderson, Vargas, Jamie McCarthy, TikTok, Jessica Maddox, she's, Maddox Organizations: Service, Business, ABC News, Police, Getty, The University of Alabama Locations: Missouri, Dee, TikTok

☆ Biden’s Race Against a Cure
▼ + stars: | 2023-11-13 | by ( The Editorial Board | ) www.wsj.com time to read: 1 min

Wonder Land: Citing the president’s age lets Democrats off the hook for the political failure of his economic policies. Images: AP/AFP/EPA/Getty Images Composite: Mark KellyThe Food and Drug Administration may soon approve two gene therapies with the potential to cure more than 100,000 Americans with debilitating sickle-cell disease. Gene therapies fix missing or defective genes. The one-time treatments have the potential to cure inherited disorders with early death sentences. The FDA has approved nine gene therapies for such diseases as Duchenne muscular dystrophy, spinal muscular atrophy and retinal dystrophy.

Persons: Mark Kelly The Organizations: AFP, EPA, Drug Administration, Medicare, Medicaid Services, FDA

Executives of some beaten-down companies, including one battered biotech name and a popular soft drinks maker, scooped up shares of their own stock in the past week. Here are the top insider buys: Energy Transfer — Executive Chairman Kelcy Warren bought one million shares Monday at $13.53 per share for a total value of $13.53 million. Shares are up more than 2% over the past month. Enstar Group CEO bought swathes of shares Wednesday, the day after the company reported its earnings for the third quarter. The stock has gained about 8% so far this year and shares are up roughly 4% over the past month.

Persons: Kelcy Warren, Dominic F, Silvester, Nigel W, Morris, Richard Barry, Verity, Douglas Ingram, Keurig Dr Pepper, Timothy P, Cofer, Dr Pepper's, Dr Pepper —, Dr Pepper, Mott's —, Enstar Organizations: CNBC Pro, —, Global, Therapeutics, Remitly, FDA, U.S . Securities, Exchange, Enstar Locations: Canada

☆ Beware these companies due to report earnings that have negative profit momentum
▼ + stars: | 2023-11-09 | by ( Lisa Kailai Han | ) www.cnbc.com time to read: +4 min

CNBC Pro screened for the S & P 500 companies still reporting next week that analysts have expressed the most caution on — by cutting their earnings estimates ahead of the reports. Analyst consensus earnings estimates are down at least 5% over the past three months. Analyst consensus earnings estimates are down at least 5% over the past six months. CTLT YTD mountain Catalent YTD chart "Catalent (CTLT) became a very noticeable laggard in the life science space about fifteen months ago. Meat producer Tyson Foods , which is due to report earnings premarket on November 13, was also among the stocks with negative earnings momentum.

Persons: Gordon Haskett, Don Bilson, Bilson, Catalent, Michael Lasser, Lasser, Morgan Stanley, BBWI, Alex Straton, Tyson Foods, — CNBC's Michael Bloom Organizations: CNBC Pro, Biotechnology, Sarepta Therapeutics, Dystrophy, UBS, 3Q, Body Locations: Columbus , Ohio, Bath

☆ Former FDA commissioner Dr. Scott Gottlieb on the future of gene therapy
▼ + stars: | 2023-11-01 | by ( ) www.cnbc.com time to read: 1 min

Share Share Article via Facebook Share Article via Twitter Share Article via LinkedIn Share Article via EmailFormer FDA commissioner Dr. Scott Gottlieb on the future of gene therapyFormer FDA commissioner Dr. Scott Gottlieb joins 'Squawk Box' to discuss the future of gene therapy, after Sarepta Therapeutics revealed new data for its Duchenne Muscular Dystrophy gene therapy fell short of expectations, the state of Covid-19, and more.

Persons: Scott Gottlieb Organizations: Former, Sarepta Therapeutics Locations: Covid

☆ Murky road ahead for Duchenne gene therapy as confirmatory trial misses main goal
▼ + stars: | 2023-10-31 | by ( Meg Tirrell | ) edition.cnn.com time to read: +4 min

Sarepta gave the one-time drug a price tag of $3.2 million, making it the second most-expensive medicine in the world. DMD affects one in 3,500 to 5,000 newborns, mostly boys because of the way it’s inherited, according to Johns Hopkins Medicine. The primary goal of the confirmatory trial was improvement on a measure known as the North Star Ambulatory Assessment, a scale of how well kids can stand, walk and perform other movements. The difference wasn’t statistically significant, meaning technically the study failed to meet its main goal. The company said no new safety issues emerged in the trial, which it called EMBARK.

Persons: Doug Ingram, ” Ingram, Sarepta, “, Dr, Sanjay Gupta, Ingram, ” Organizations: CNN, US Food and Drug Administration, Sarepta Therapeutics, FDA, Johns Hopkins Medicine, North Star, CNN Health

☆ The life and career of Lululemon founder Chip Wilson
▼ + stars: | 2023-10-27 | by ( Jordan Hart | Jennifer Ortakales Dawkins | ) www.businessinsider.com time to read: +2 min

Chip Wilson founded Lululemon Athletica Inc. in 1997 after selling his first apparel company. Wilson has lived with a rare muscular disease for over 30 years. AdvertisementAdvertisementLululemon 's billionaire founder Chip Wilson has been the center of both acclaim and controversy. He spends part of his fortune on philanthropy and researching a cure for the rare muscular disease called facioscapulohumeral muscular dystrophy, which he was diagnosed with at 32 years old. Here's everything we know about Wilson's life and career as the founder and former CEO of Lululemon.

Persons: Chip Wilson, Wilson, —, women's Organizations: Lululemon Athletica, Service, Bloomberg

☆ These stocks are trying to solve a major shortcoming of weight loss drugs: Shrinking muscle mass
▼ + stars: | 2023-10-22 | by ( Christina Cheddar Berk | ) www.cnbc.com time to read: +9 min

One of the downsides of taking new anti-obesity medications is that patients tend to lose lean muscle mass along with body fat. "This is a major shortcoming in terms of not just quality of life for the patients who have to lose lean muscle mass, but there's actually room to improve on the weight loss and there [are] other metabolic benefits to improving your lean muscle mass ... [including] you might have better durability of weight loss." The drug is being tested in a phase 2b study alongside semaglutide to protect against loss of muscle mass. Until then, patients will need to watch what they eat and focus on exercise just as they would on any other weight loss regimen. When that occurs, muscle loss will be an even bigger issue than it is now.

Persons: Piper Sandler, Allison Bratzel, there's, Bratzel, Versanis, Eli Lilly, Eli Lilly's Mounjaro, they're, Lydia Alexander, Alexander, Sandler's Bratzel, incretins, Biohaven, Nestle, Ulf Mark Schneider, Jeff Jonas, Jonas, Stephens, Jim Salera, Atkins, Morgan Stanley, — CNBC's Michael Bloom Organizations: Wall, Nordisk's Ozempic, Obesity Medicine Association, Bristol Myers Squibb, Nestle, Gabelli, Quest, Foods Locations: Swiss

☆ The founder of Lululemon is spending $100 million to try to beat the super rare disease that's destroying his muscles
▼ + stars: | 2023-10-22 | by ( Lakshmi Varanasi | ) www.businessinsider.com time to read: +3 min

Lululemon founder Chip Wilson is battling FSHD, a rare form of muscular dystrophy. He's investing $100 million into finding a cure through his venture philanthropy fund, Solve FSHD. AdvertisementAdvertisementLululemon founder Chip Wilson has been battling a rare muscular disease for the past several decades, and he's funneling $100 million of his multibillion net worth into finding a cure. Wilson suffers from an even more uncommon form called FSHD2 that impacts just 5% of those with the disease, Bloomberg noted. Solve FSHD did not immediately respond to Insider's request for a comment.

Persons: Chip Wilson, FSHD, He's, Wilson Organizations: Morning, Bloomberg, FSHD Society, China's, Anta, Products, Amer Sports

☆ A disabled Israeli teenager with muscular dystrophy is a hostage held by Hamas in Gaza after she was abducted from the Supernova music festival
▼ + stars: | 2023-10-14 | by ( Rebecca Rommen | ) www.businessinsider.com time to read: +3 min

Hamas militants took a disabled 17-year-old hostage who attended the Supernova music festival. AdvertisementAdvertisementA disabled 17-year-old girl, Rut Perez, is among the 150 hostages abducted by Hamas militants, The Times of London reports. The vulnerable teenager, who has muscular dystrophy, was attending the Supernova festival near the Gaza border when Hamas militants massacred over 260 attendees. Rut attended the electronic music festival with her sister, Yamit, and her father. "I want to believe there are good people in this world and they will help us get our family back," Yamit told The Times.

Persons: Perez, —, Yamit, Israel Organizations: Supernova, Service, Times, Palestinian, UN Locations: Gaza, London, Tel Aviv

☆ Stocks making the biggest moves midday: General Motors, Stellantis, Planet Fitness, Adobe and more
▼ + stars: | 2023-09-15 | by ( Alex Harring | ) www.cnbc.com time to read: +4 min

Nucor — The steelmaker fell 6.1% after offering worse-than-expected guidance for third-quarter earnings, with the company pointing to pricing and volume challenges. Core & Main — The infrastructure stock retreated 4.1% a day after it announced a secondary stock offering. The bank said Apellis has a favorable risk/reward ahead of third-quarter earnings. The Wall Street firm said the resumption of loan repayments introduce bookings risk to food delivery. The Wall Street firm said its pessimistic outlook was changing despite recent underperformance in the reinsurance space.

Persons: Stellantis, Chris Rondeau, Rondeau, Craig Benson, Nucor, Raymond James, Needham, Charles Shi, Lam, Goldman Sachs, Apellis, MoffettNathanson, Estée Lauder, Goldman, Yun Li, Jesse Pound, Samantha Subin, Pia Singh, Brian Evans, Lisa Kailai Han Organizations: UAW, General Motors Wentzville, General Motors, Ford, United Auto Workers began, Workers, CNBC, LSEG, PTC Therapeutics, Medicines Agency's, Medicinal Products, Arm, Investment, Bloomberg News, Apple, Dexcom, ASML Holding, Lam Research, Taiwan Semiconductor, Adobe, Bank of America, JPMorgan, Apellis Pharmaceuticals, Waste Systems Locations: Wentzville , Missouri, New Hampshire, Wells, underperform

☆ Analysts love these Nasdaq stocks as benchmark wraps up best first half since 1983
▼ + stars: | 2023-06-27 | by ( Brian Evans | ) www.cnbc.com time to read: +2 min

The tech-heavy index has soared nearly 29% this year and is currently on pace for its best first half since 1983. That year, the Nasdaq rallied 37.1% in the first half and finished 1983 up nearly 20%. Their average price target implies about 34% upside compared with current levels. Three-quarters of analysts covering the stock rate it a buy, and the average price target indicates 30% upside over the next 12 months. Of the 14 analysts covering the stock, 71% rate it a buy, while the average analyst price target implies upside of nearly 64%.

Persons: Ambrx, FANG, Canaccord Genuity Organizations: Nasdaq, Apple, Google, Nvidia, CNBC Pro, Diamondback Energy, Mobileye, Sarepta Therapeutics, U.S . Food, Drug Administration Locations: Texas, U.S

☆ Sarepta slumps as concerns emerge around upcoming gene therapy data
▼ + stars: | 2023-06-23 | by ( ) www.reuters.com time to read: +2 min

June 23 (Reuters) - Sarepta Therapeutics shares (SRPT.O) slumped 11% on Friday as some analysts voiced concerns that upcoming confirmatory trial data for its gene therapy to treat Duchenne muscular dystrophy (DMD) may not be enough to secure approval for expanded use. The U.S. health regulator had on Thursday granted Sarepta's Elevidys therapy accelerated approval to treat DMD patients aged between 4 and 5 years who can walk, contrary to the company's application for all patients who can walk. Sarepta's gene therapy is the first of its kind for DMD, an inherited progressive muscle-wasting disorder that almost always affects young boys. Elevidys, a one-time treatment, is expected to change the way that DMD patients are treated as current therapies require regular use. William Blair's Tim Lugo called the treatment "transformational for Duchenne's patients" and "a significant opportunity for Sarepta".

Persons: Sarepta's, Gavin Clark, Gartner, William Blair's Tim Lugo, Leroy Leo, Khushi, Devika Organizations: Sarepta Therapeutics, Food and Drug, BioMarin Pharmaceuticals, Thomson Locations: U.S, Bengaluru

☆ First Gene Therapy for Muscular Dystrophy Approved
▼ + stars: | 2023-06-22 | by ( Liz Essley Whyte | ) www.wsj.com time to read: 1 min

This copy is for your personal, non-commercial use only. Distribution and use of this material are governed by our Subscriber Agreement and by copyright law. For non-personal use or to order multiple copies, please contact Dow Jones Reprints at 1-800-843-0008 or visit www.djreprints.com. https://www.wsj.com/articles/first-gene-therapy-for-muscular-dystrophy-approved-34860988

Persons: Dow Jones

☆ The Price of First Gene Therapy for Muscular Dystrophy: $3.2 Million
▼ + stars: | 2023-06-22 | by ( Liz Essley Whyte | ) www.wsj.com time to read: 1 min

Persons: Dow Jones

☆ FibroGen's neuromuscular disorder drug fails in late-stage study
▼ + stars: | 2023-06-07 | by ( ) www.reuters.com time to read: 1 min

June 7 (Reuters) - FibroGen Inc (FGEN.O) said on Wednesday its drug to treat a type of neuromuscular disorder failed to meet the main goal in a late-stage study, sending the company's shares down about 14% in premarket trading. The treatment was generally safe and well tolerated for patients with Duchenne muscular dystrophy (DMD). DMD is a progressive degenerative disorder that hampers muscle movement and affects one in 3,600 newborn boys, most of whom die by the age of 30. Reporting by Sriparna Roy in Bengaluru; Editing by Shilpi Majumdar and Shounak DasguptaOur Standards: The Thomson Reuters Trust Principles.

Persons: Sriparna Roy, Shilpi Majumdar, Shounak Organizations: FibroGen, Thomson Locations: Bengaluru

☆ Catalent cuts annual forecast hurt by operational challenges
▼ + stars: | 2023-05-19 | by ( ) www.reuters.com time to read: +2 min

May 19 (Reuters) - Catalent Inc (CTLT.N) cut its full-year net revenue and profit forecast on Friday, reflecting operational challenges and higher-than-expected costs after it flagged an over $400 million hit to both its annual sales and core profit forecasts earlier this month. The contract drug manufacturer now sees its full-year revenue in the range of $4.25 billion to $4.35 billion compared with its prior forecast of $4.63 billion to $4.88 billion. Catalent said it now sees adjusted net income for the year in the range of $187 million to $228 million, compared with the previous forecast of $567 million to $648 million. Shares of Catalent reversed losses to be up 7.7% at $34.63 before the bell. Reporting by Bhanvi Satija, Sriparna Roy and Pratik Jain in Bengaluru; Editing by Krishna Chandra EluriOur Standards: The Thomson Reuters Trust Principles.

☆ Gene-Therapy Investors Have a Powerful Ally: Patients
▼ + stars: | 2023-05-16 | by ( David Wainer | ) www.wsj.com time to read: 1 min

Sarepta’s stock jumped after an FDA panel voted in favor of a drug to treat Duchenne muscular dystrophy. Photo: Kristoffer Tripplaar/Sipa USA/Associated PressImagine for a moment that you are an expert at a Food and Drug Administration advisory panel meeting trying to decide whether to vote in favor of a drug for a rare disease that robs boys of their ability to walk, eventually killing many of them by the time they reach young adulthood. During the meeting, you hear testimonials from doctors and patients, including a video showing a boy riding his bike years after receiving the therapy for the disease, known as Duchenne muscular dystrophy. At the same meeting, you also hear from FDA staff who warn you that the studies conducted by Sarepta Therapeutics don’t provide clear evidence that the drug is beneficial. They also highlight some safety concerns.

The caller was an Israeli officer who ordered them to evacuate the house five minutes before they bombed it. The cousin, Hussam Nabhan, 45, tried to stall with the officer, telling him the house included disabled people, but it was all in vain, he said. "The house was the girls' shelter, they had got a disabled toilet, wheelchairs, a bed to sleep. "How am I going to carry her after the wheeler was gone, also the (healthy) mattresses were gone," she added. Its borders are sealed off by neighbouring Israel and Egypt, citing security concerns with Hamas, which runs the territory.

☆ Sarepta surges after FDA panel backs Duchenne gene therapy
▼ + stars: | 2023-05-15 | by ( ) www.reuters.com time to read: +2 min

May 15 (Reuters) - Shares of Sarepta Therapeutics (SRPT.O) soared 29% premarket on Monday as a backing by the U.S. health regulator's advisers increased the certainty of an accelerated approval for the company's gene therapy for a muscle-wasting genetic disorder. Eight of the 14 advisers to the Food and Drug Administration late Friday voted that the company had enough data to support an accelerated approval for its gene therapy for Duchenne muscular dystrophy (DMD). The vote came after FDA staff had earlier last week voiced concerns that the data from the company's mid-stage trial lacked "unambiguous evidence" about benefits from the therapy. TD Cowen analyst Ritu Baral expects an approval for the therapy, especially considering the FDA leadership's interest in promoting biomarker-based accelerated approvals for gene therapies. The FDA, often follows the advice of its expert advisers but is not obligated to do so, is slated to make a decision on accelerated approval by May 29.

☆ FDA board votes to approve gene therapy for Duchenne muscular dystrophy
▼ + stars: | 2023-05-15 | by ( ) www.cnbc.com time to read: 1 min

In this videoShare Share Article via Facebook Share Article via Twitter Share Article via LinkedIn Share Article via EmailFDA board votes to approve gene therapy for Duchenne muscular dystrophyDavid Nierengarten, Wedbush Securities managing director, joins 'Closing Bell Overtime' to discuss Sarepta shares after the FDA panel votes to approve gene therapy for Duchenne muscular dystrophy.

☆ FDA reviewers raise doubts over data on Sarepta's gene therapy
▼ + stars: | 2023-05-10 | by ( Leroy Leo | ) www.reuters.com time to read: +2 min

May 10 (Reuters) - The U.S. Food and Drug Administration's staff reviewers raised concerns over limited data on Sarepta Therapeutics Inc's (SRPT.O) gene therapy for a muscle-wasting disorder, ahead of a meeting of the agency's advisers on Friday. The agency's staff on Wednesday highlighted several issues with the data and said that existing studies did not provide "unambigious evidence" that the gene therapy will benefit patients with Duchenne muscular dystrophy. The FDA also highlighted that DMD, a rare disorder that mainly affects young boys, is an urgent unmet medical need. The advisory committee's vote on Friday and an eventual decision by the FDA is seen as a test of the agency's accelerated approval pathway for gene therapies. Sarepta has three drugs available for the disease for people with different types of genetic mutations but they have to be given as weekly intravenous infusion, while the experimental gene therapy is a one-time treatment.

☆ Sarepta shares tumble as concerns mount over FDA approval on gene therapy
▼ + stars: | 2023-04-13 | by ( ) www.cnbc.com time to read: 1 min

Share Share Article via Facebook Share Article via Twitter Share Article via LinkedIn Share Article via EmailSarepta shares tumble as concerns mount over FDA approval on gene therapyCNBC's Meg Tirrell joins 'Power Lunch' to discuss the Sarepta's gene therapy for muscular dystrophy, and why the stock is taking a tumble over doubts around Sarepta receiving FDA approval.

☆ Sarepta drops as report says FDA almost rejected under-review gene therapy
▼ + stars: | 2023-04-13 | by ( ) www.reuters.com time to read: +1 min

April 13 (Reuters) - Sarepta Therapeutics Inc's shares (SRPT.O) fell more than 6% on Thursday as Stat News reported that the U.S. health regulator's staff were inclined to reject the company's gene therapy which is currently under review. Some staff at the U.S. Food and Drug Administration (FDA) had reached a "non-binding conclusion" that Sarepta's gene therapy should not be given approval, prompting FDA official Peter Marks to intervene and instead schedule an advisory meeting on May 12, Stat News reported, citing sources. The company is seeking approval for its gene therapy for treating a rare muscle-wasting disorder called Duchenne muscular dystrophy under the FDA's accelerated pathway. Brokerage William Blair said it still sees a "likely" approval for the therapy. Shares of Catalent Inc (CTLT.N), which holds the contract manufacturing rights for the therapy, fell as much as 5.5% in early trade.

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