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The FDA has approved a new gene therapy to treat hemophilia B, a genetic bleeding disorder. The drugmaker CSL Behring set a $3.5 million price for the one-time treatment. Lackey is learning to live with a severe case of hemophilia B, a rare genetic condition that keeps blood from clotting properly. But a critical unknown in judging CSL's $3.5 million price is the durability of the treatment. Lojewski said the $3.5 million price wasn't guided by other gene-therapy prices.
The FDA has approved a new gene therapy to treat hemophilia B, a genetic bleeding disorder. Hemophilia patients told Insider they're excited about the new drug but worried about the price. Lackey is learning to live with a severe case of hemophilia B, a rare genetic condition that keeps blood from clotting properly. While the treatment breaks ground as the first approved gene therapy for hemophilia, its price is also unprecedented. Lojewski said the $3.5 million price wasn't guided by other gene-therapy prices.
She is the CEO of the $12 billion biotech United Therapeutics and founder of SiriusXM. Her company, the $12 billion Maryland-based biotech United Therapeutics, has already put gene-edited pig hearts, lungs, and kidneys into humans in clinical studies. Rothblatt previously founded SiriusXM before starting United TherapeuticsUnited TherapeuticsThe organ research is far from the first moonshot in Rothblatt's career. After that compound's owner, the pharma giant GSK, declined to test it against PAH, Rothblatt founded United Therapeutics in 1996 and bought the rights to the molecule. That drug helped save Jenesis, and United Therapeutics now sells a suite of PAH treatments that have helped over 12,000 patients.
There aren't any savings account limits if you're applying for Social Security Disability Insurance. Here's what you need to know about your savings options if you'd like to receive disability benefits. Can you have a savings account if you receive Social Security disability benefits? Savings account options if you receive Social Security disability benefitsSavings accountYou can open regular savings account at a brick-and-mortar or online bank. If they plan on using it as an emergency reserve savings account, they may want to keep it in cash," advises Dunn.
Share Share Article via Facebook Share Article via Twitter Share Article via LinkedIn Share Article via EmailChina protests send global stocks lower as experts see Covid disruption persistingGlobal stocks moved lower after rare protests erupted across China over the country's stringent zero-Covid policy — the Dow declined 497.57 points while the S&P 500 dropped by 62.18 points. Six experts discuss the impact on U.S. markets.
Lee County suffered none of the state’s 123 Irma deaths. That experience influenced many people’s decisions not to flee Hurricane Ian. “Hurricane Ian is going to be the strongest outreach messaging for southwest Florida ever,” said Gleason, the Charlotte County spokesman. “When you live through it, then that message sinks in deep and forever.”A view of the Matanzas Pass side of Estero Island, home to Fort Myers Beach, Fla. Fort Myers Beach is expected to take years to rebuild. Thomas Simonetti for NBC NewsMichael Yost's two closest friends in Fort Myers Beach drowned in Hurricane Ian.
In June, Charm said it raised $50 million, valuing the firm at $100 million to $150 million. Charm has raised $50 million from top investorsDemis Hassabis, the CEO and a cofounder of DeepMind Technologies. In the spring, Aithani raised the $37 million million round that was announced in June, with investors like Khosla Ventures and General Catalyst joining OrbiMed and F-Prime Capital. The raise values Charm at between $100 million and $150 million, Aithani said, and brings the company's total funding to $50 million. This article was corrected on August 19 to show that Charm has raised two rounds of funding totaling $50 million.
NBA roundup: Kyrie Irving returns, helps Nets top Grizzlies
  + stars: | 2022-11-21 | by ( ) www.reuters.com   time to read: +7 min
Dillon Brooks led all scorers with 31 points but shot 13 of 30 as the Grizzlies saw their seven-game winning streak over Brooklyn halted. John Konchar added 16 while Steven Adams contributed 15 and 10 rebounds as the Grizzlies shot 43.4 percent. Bruce Brown scored 12 points and DeAndre Jordan had 17 rebounds for the short-handed Nuggets. Six Cavs scored at least 11 points with Lamar Stevens posting 11 and Evan Mobley recording a double-double of 15 and 10 rebounds. KJ Martin scored 12 points off the bench.
The biotech FogPharma has raised $178 million in a Series D round. The longtime Harvard chemist Greg Verdine founded Fog to tackle "undruggable" cancers. Fog's drugs, known as polypeptides or short fragments of proteins, blur the line between small molecules and biologics, sharing characteristics of both types of medicines. Verdine calls it "the magic structure" for Fog's drugs. "I thought he'd be able to find things that bind, but I was very skeptical that he could turn them into drugs," Klausner told Insider.
What happens to inflation in 2023?
  + stars: | 2022-11-21 | by ( Noah Higgins-Dunn | Jeff Morganteen | Jeff Cox | ) www.cnbc.com   time to read: +1 min
There are hints that the worst of the U.S.'s bout with inflation may be in the past. The consumer price index, a widely watched inflation gauge, came in at 7.7% in October when compared with a year earlier. Inflation can be very hard to predict," Kevin Kliesen, business economist and research officer at the Federal Reserve Bank of St. Louis, told CNBC in an interview. However, any potential downturn is expected to be mild, The Conference Board CEO Steve Odland told CNBC. This could be a much less painful experience with the Fed trying to tame inflation than it has been in the past," Odland said.
There are hints that the worst of the U.S.'s bout with inflation may be in the past. The consumer price index, a widely watched inflation gauge, came in at 7.7% in October when compared with a year earlier. While that was still well above the Federal Reserves' 2% target, it did clock in below Wall Street's expectations. However, any potential downturn is expected to be mild, The Conference Board CEO Steve Odland told CNBC. This could be a much less painful experience with the Fed trying to tame inflation than it has been in the past."
Several Biden aides and advisers, speaking on condition of anonymity, gave their assessment of how the president and his team view Trump's entry into the race. Trump made his announcement on Tuesday night as he sought to get a jump on potential rivals for the Republican nomination. Biden, who defeated Trump in the contentious 2020 election, thus far is remaining mum publicly. Biden defeated Trump by more than 7 million in the nationwide popular vote tally and by a margin of 306 to 232 in the state-by-state Electoral College that determines the outcome of presidential elections. A few hours before Trump's announcement, the White House launched a new webpage highlighting the Biden administration's achievements from creating manufacturing jobs and lowering drug prices for seniors to gun reform.
A Baltimore-based biotech startup has raised $56 million to develop a cancer blood test. Haystack Oncology has raised $56 million in a Series A round to continue development of its cancer blood test in 2023, the startup said Wednesday. The Baltimore-based biotech says it has a blood test that can find tiny, lingering traces of cancer in patients who've already had cancer and received treatment. Those cancerous cells shed small pieces of their DNA into the bloodstream, and Haystack's test detects those molecules. Haystack's blood test, called a liquid biopsy, can find signs of cancer that aren't detectable by imaging like CT scans.
President Biden's student loan forgiveness plan is currently paused after two court rulings. Over the past week, courts handed Biden's student-debt relief plan two major blows. That decision was in favor of the six Republican-led states who sued the debt relief, arguing it would hurt their states' tax revenues. "The AFL-CIO is extremely disappointed in the partisan legal effort to shut down the Biden administration's life-changing student loan relief. The federation's president, Liz Shuler, called on Biden to cancel student debt in the spring.
"I know I'm a cockeyed optimist," Biden told reporters in Cambodia, referencing a sunny song from the musical South Pacific, "but I'm not surprised by the turnout." A Democratic victory in a Georgia Senate runoff on Dec. 6 would give the party outright majority control, bolstering its sway over committees, bills, and judicial picks. As of Sunday evening, Republicans had won 211 seats and the Democrats 206, with 218 needed for a majority. World leaders at an East Asia summit of ASEAN nations told Biden they are closely following the midterms results, national security adviser Jake Sullivan told reporters aboard Air Force One. He has said he will invite Republicans and Democrats to the White House once he returns.
A federal judge in Texas struck down Biden's student-loan forgiveness on Thursday. Now, however, the Education Department has stopped accepting applications. "We are disappointed in the decision of the Texas court to block loan relief moving forward," he added. "Amidst efforts to block our debt relief program, we are not standing down." Where student-loan forgiveness goes from hereShortly after the Texas ruling, the Education Department said the Justice Department had filed an appeal of the court's decision, which will go to the conservative 5th Circuit.
CompaniesCompanies Law firms Gibson, Dunn & Crutcher Llp Follow(Reuters) - Gibson, Dunn & Crutcher said Monday that Gustav Eyler, who was a top U.S. Department of Justice consumer protection official, has joined the firm as a Washington, D.C.-based partner. Eyler, who practiced as a litigation associate at Los Angeles-founded Gibson Dunn earlier in his career, served for five years as director of the consumer protection branch of the Justice Department's civil division, where he oversaw more than 250 prosecutors and staff, the firm said. Gibson Dunn has made other recent hires to its Washington office, including two last week. At Gibson Dunn, Eyler said he plans to help clients facing government investigations, enforcement actions and related consumer class actions involving the life sciences, healthcare and consumer products sectors, and work with clients on data privacy matters. Read more:Law firm Steptoe hires U.S. Justice Dept senior antitrust lawyerJustice official to lead Jenner's congressional investigations teamOur Standards: The Thomson Reuters Trust Principles.
President Joe Biden on Sunday touted the results of the midterm elections, with Democrats projected to maintain control of the U.S. Senate following Democratic Sen. Catherine Cortez Masto’s narrow defeat of Republican Adam Laxalt to win re-election in Nevada. Democrats defeated several candidates backed by former President Donald Trump to hold onto at least 50 seats come 2023. Democrats, meanwhile, joined Biden in celebrating their election wins, with some also pointing to Trump as the reason why they outperformed Republicans. Their candidates were talking about lack of democracy," Senate Majority Leader Chuck Schumer told reporters at a news conference in New York on Sunday. And they have produced a great result.”On “Meet the Press," Anita Dunn, senior adviser to the president, said: “It’s very clear what President Biden and the Democratic Party are for.
Share this -Link copiedWisconsin Senate race too close to call Wisconsin's Senate race between GOP Sen. Ron Johnson and Democrat Mandela Barnes is too close to call, NBC News says. Vance wins Ohio Senate race, defeating Democrat Tim Ryan, NBC News projects COLUMBUS, Ohio — J.D. Ohio Republican Senate candidate J.D. Vance is leading The Senate race in Ohio is too early to call, NBC News says, but Republican candidate J.D. Share this -Link copiedGeorgia Senate race too close to call Georgia's Senate race is too close to call about three hours after polls closed at 7 p.m.
CMA Awards 2022: See the full list of winners
  + stars: | 2022-11-09 | by ( Dan Heching | ) edition.cnn.com   time to read: +3 min
CNN —Country music threw a party on Wednesday night as the CMA Awards were presented. First-time nominee Lainey Wilson topped the list of nominees going into the show and ended the night with major wins, including new artist of the year and female vocalist of the year. The emotional Wilson declared in one acceptance speech: “I know I’m new to a lot of folks, but I won’t let y’all all down. I promise you.”Luke Combs also had a big night, taking the stage both as a performer and winner. Other performers included Kelly Clarkson, who performed with Kelsea Ballerini and Carly Pearce, and Luke Bryan, who pulled double duty as host alongside Peyton Manning.
As a result, political observers say, public school funding is effectively on the ballot Tuesday. “These groups have been demonizing what is being taught in public schools, and that’s the fastest way to erode faith that public schools work,” Rottinghaus said. (Abbott publicly came out in support of private school vouchers two months after winning the primary with 66.5% of the vote.) Greg Abbott in the GOP primary, campaigned in support of private school vouchers. “I will never support vouchers.”Rep. John Bucy III said he will continue to oppose private school vouchers.
Ovechkin's power-play goal at 8:55 of the second period came in his 1,287th game with Washington, snapping a tie with Hockey Hall of Famer Gordie Howe. Anthony Mantha set up that goal and scored one of his own before the Coyotes stormed back. Ritchie's first goal of the game sailed just under the crossbar and over the goal line to forge a tie at 9:45. Zetterlund's second goal of the game also snapped New Jersey's eight-game losing streak to Calgary. Gourde finished with a goal and two assists, Tanev added an assist, and Vince Dunn also scored for the Kraken.
WASHINGTON — Federal prosecutors rested their case against Oath Keepers founder Stewart Rhodes and four other members of the far-right organization on Wednesday without calling three cooperating defendants who pleaded guilty to seditious conspiracy. But the government did not call for three members of the group who pleaded guilty to seditious conspiracy — Joshua James, Brian Ulrich and William Todd Wilson — before resting their case. Stewart Rhodes, founder of the Oath Keepers, speaks during a rally outside the White House in Washington on Jun. Susan Walsh / AP fileProsecutors may have decided that presenting any of the three defendants who pleaded guilty to seditious conspiracy would be too much of a risk on cross examination. Prosecutors also can't explicitly argue that because a witness pleaded guilty to seditious conspiracy that the defendants are guilty of the same charge, which limits the value of their testimony.
It would be the first new pill to treat uncomplicated UTIs in 20 years, GSK said. The drug, called gepotidacin, could become the first new oral antibiotic for uncomplicated UTIs in more than two decades, according to the company. GSK's drug was tested in uncomplicated UTIs, which are restricted to the the bladder and urethra and are generally less serious than complicated UTIs. On Thursday, GSK said two studies enrolled over 3,000 patients with uncomplicated UTIs. GSK, on the other hand, paid $66 million in September to license another antibiotic program being tested to treat complicated UTIs.
WASHINGTON, Nov 2 (Reuters) - President Joe Biden will say on Wednesday that threats by some Republican candidates to refuse to accept election results if they lose is a "path to chaos" and will urge voters to reject election deniers. Biden will speak at a 7 p.m./2300 GMT Democratic National Committee event at Washington's Union Station near Capitol Hill. The Democratic National Committee said Biden would "address the threat of election deniers and those who seek to undermine faith in voting and democracy; and the stakes for our democracy in next week’s election." "This is an inflection point," she told a regular White House briefing. Some 44% said they are concerned that the U.S. election is rigged, including 28% of Democrats and 62% of Republicans.
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