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First gene therapy for deadly form of muscular dystrophy gets FDA approval for young kids

Published Sat, 22 Feb 2025 04:42:59 GMT

By MATTHEW PERRONE (AP Health Writer)WASHINGTON (AP) — The first gene therapy for a deadly form of muscular dystrophy received preliminary U.S. approval on Thursday despite concerns from some government scientists about the treatment’s ability to help boys with the inherited disease.The Food and Drug Administration approval provides a new option for some patients with Duchenne muscular dystrophy, a rare muscle-wasting disease that causes weakness, loss of mobility and early death. It almost always affects males.Drugmaker Sarepta Therapeutics said it would charge $3.2 million for the one-time treatment, slightly less than a $3.5 million gene therapy for hemophilia launched last year. Like most medicines in the U.S., the cost will be mostly paid by insurers — not patients — including private plans and government programs.The FDA OK’d the treatment only for children ages 4 and 5, based on study results showing the therapy helped produce a protein needed fo...

Live updates | Titanic salvage rights owner mourns expert killed aboard Titan

Published Sat, 22 Feb 2025 04:42:59 GMT

By The Associated PressFollow along for live updates on the submersible that vanished while taking five people down to the wreck of the Titanic.___TITANIC SALVAGE RIGHTS OWNER MOURNS LOSS OF SHIPWRECK EXPERTRMS Titanic, Inc., the company that owns the salvage rights to the Titanic shipwreck, is mourning the loss of Titanic expert Paul-Henri Nargeolet, who was among five people killed aboard the Titan submersible when it imploded this week.Nargeolet — known as “PH” — was a long-term employee of the company.“The maritime world has lost an iconic and inspirational leader in deep-sea exploration, and we have lost a dear and treasured friend,” the company said in a statement Thursday.Friend and former colleague Matthew Tulloch said Nargeolet loved his work from the time they collaborated in the 1990s up until Nargeolet’s death.“I never got the impression that he was looking forward to retirement,” Tulloch said with a small laugh. R...

Yankees Notebook: Giancarlo Stanton returns to OF as Aaron Judge eyes end-of-week baseball activities

Published Sat, 22 Feb 2025 04:42:59 GMT

After a few days of consideration, Giancarlo Stanton finally returned to the outfield on Thursday against the Mariners.Stanton, penciled into right, hasn’t played the outfield since April 12 or since coming back from a hamstring injury on June 2. His defensive workload has been a talking point throughout his Yankees career, but Aaron Boone is not sure how often he’ll use the slugger in the field.“Hopefully, it remains in place throughout the year,” Boone said, adding that he’ll discuss it with Stanton. “It’s kind of something that we’ll tackle week by week and day by day.”Stanton has said in the past that he believes playing the outfield helps his swing, and Boone has said that it’s good for the oft-injured player to stay active.But with Stanton in the middle of a slump, Boone has also tried to get ahead of narratives should the former MVP start to hit now that he’s using his glove again.“If he goes out and bang...

Orioles catcher Adley Rutschman advances to next phase in MLB’s All-Star voting, with chance to start for AL

Published Sat, 22 Feb 2025 04:42:59 GMT

The first phase of All-Star fan voting ended Thursday, and Orioles catcher Adley Rutschman is one step closer to potentially starting the Midsummer Classic for the American League.Rutschman will face Texas Rangers catcher Jonah Heim in the second phase of voting, during which fans will determine who will start July 11 at Seattle’s T-Mobile Park.Although Rutschman led Heim by a significant margin in the latest update, the vote totals from the first phase don’t carry over to the second. Voting will restart at noon Monday and end at noon Thursday, with fans allowed to vote once per 24-hour period.Rutschman, 25, is slashing .272/.382/.425 — good for an .808 OPS — with 10 home runs, 32 RBIs and an AL-leading 49 walks. Heim, 27, has the same number of home runs as Rutschman (10) with 22 more RBIs as part of the Rangers’ MLB-best offense. Heim’s .792 OPS is slightly worse than Rutschman’s, but his 2.6 WAR ranks best among AL catchers.Rutschman, in ...

North Carolina GOP legislators making changes to new abortion law days before enforcement

Published Sat, 22 Feb 2025 04:42:59 GMT

RALEIGH, N.C. (AP) — North Carolina Republican legislators rolled out on Thursday adjustments to the state’s new abortion restrictions that are set to take effect in days, addressing some provisions that litigation seeking to block the law’s enforcement calls confusing and inconsistent.GOP senators said the changes offered on the Senate floor were small, designed to affirm the intent of the measure enacted last month over Democratic Gov. Roy Cooper’s veto that in part would ban starting July 1 nearly all abortions after 12 weeks of pregnancy. “We very much see it as something that is technical and clarifying,” Sen. Ralph Hise, a Mitchell County Republican, told reporters after the Senate voted for the abortion alterations, tacked on to another pending state health agency bill. “It makes no substantive changes.”But the language appears to attempt to thwart a federal lawsuit filed last week by Planned Parenthood South Atlantic and a doctor who performs abortions that...

Families detail stress, terror and sadness after Nashville school shooting in court documents

Published Sat, 22 Feb 2025 04:42:59 GMT

NASHVILLE, Tenn. (AP) — Nearly three months after a shooter killed six people at her daughter’s school in Nashville, Katherine Heath watches the third-grader lie on her husband’s chest whenever her child has a “sad day.”After losing multiple classmates, Heath’s daughter is now visibly withdrawn, attending counseling, and learning to live in a world without her best friend.“She will come to my husband and just lay on his chest. He will say something like ‘It’s been a hard month baby,’” Heath wrote in a recent court filing. “And she’ll just faintly reply `yea.’”Heath and more than a dozen other parents wrote about life after the March 27 shooting at The Covenant School, offering images of their still-shaken children to compel a judge not to allow the release of the shooter’s writings and other documents. Audrey Hale, a 28-year former Covenant student, indiscriminately opened fire at the private Christian elementary school in March, killing three children ...

Mexico’s top court again overrules president on electoral reform

Published Sat, 22 Feb 2025 04:42:59 GMT

MEXICO CITY (AP) — Mexico’s Supreme Court on Thursday overturned another set of electoral law changes favored by President Andrés Manuel López Obrador. The 9-2 ruling is likely to further fan the growing dispute between the president and the courts, which have blocked some of his key legal overhauls, including a related change in May. The justices said López Obrador’s Morena party changed the electoral legislation after it was approved in Congress.The reforms would have reduced the spending and size of the country’s independent National Electoral Institute, and limit its authority to oversee political parties and candidates. López Obrador has accused the high court of overstepping its authority by judging whether Congress followed its own rules.In May, the court overturned another part of the electoral changes because Congress rushed the measure through to a vote without debate, or even time to read it.López Obrador has a habit of demanding legislators approve bills they often...

Trump, DeSantis interviews show Fox influence on GOP field still strong despite troubled year

Published Sat, 22 Feb 2025 04:42:59 GMT

NEW YORK (AP) — The two leading GOP presidential contenders had very different interview experiences with Fox News in the past week — each an illustration of the influence that even a damaged Fox has over the Republican nominating process.Donald Trump’s interview with Bret Baier, which aired in two parts Monday and Tuesday, was meaty and newsworthy. Baier pressed the former president about his indictment on hoarding confidential documents and pushed back on Trump’s false claims about the 2020 election.Ron DeSantis’ session with Trump’s former press secretary, Kayleigh McEnany, on Wednesday was far softer.Their appearances alone are evidence that Fox, weakened financially by the $787 million settlement over defamation charges reached with Dominion Voting Systems and suffering in the ratings following Tucker Carlson’s firing, remains the media kingmaker for Republicans who want to be president.After the first part of his Trump interview aired, Fox announc...

Montreal police chief rejects street check moratorium despite racial profiling data

Published Sat, 22 Feb 2025 04:42:59 GMT

Montreal’s police chief is refusing to impose a moratorium on street checks despite a second independent report concluding that racialized people are disproportionately targeted by the practice.Fady Dagher made the comments Thursday after he released an independent report by researchers from several Quebec universities who recommended he temporarily suspend random police stops. A moratorium would be purely symbolic, he said, adding that he preferred “constant small steps” to address racial profiling and ensure police street checks respect residents’ rights.“I know I’m going to create some disappointment,” the police chief said of his decision to reject the moratorium. “But I don’t want to announce a symbolic measure. I want to solve the deep problem at the roots.”Researchers looked at 2021 data and found that Indigenous Montrealers were six times more likely to be stopped than were white people in the city. The data showed ...

Canopy Growth reports $648 million net loss in Q4 as it parts way with BioSteel staff

Published Sat, 22 Feb 2025 04:42:59 GMT

Canopy Growth Corp.’s chief executive said the company has made management changes and parted ways with some staff as it continues to review its BioSteel business after uncovering “material misstatements” in the sports drink unit’s previous financial filings.“Based on the results of the review, we’ll be implementing several remedial actions to strengthen our controls for the BioSteel business,” David Klein, chief executive of the Smiths Falls, Ont.-based cannabis company, said on a Thursday call with analysts.“We felt it was important to act swiftly to provide stability to the business at this pivotal time, so to this effect, we have exited several members of the BioSteel leadership team and are considering all legal remedies available to us including litigation to recover damages and costs associated with and resulting from the findings of the BioSteel review.”His remarks came after Canopy promised in May to refile three of its ...