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FDA's Bespoke Pathway To Focus on Gene Editing and RNA-Based Treatments for Rare Diseases

FDA's Bespoke Pathway To Focus on Gene Editing and RNA-Based Treatments for Rare Diseases

The FDA has introduced a draft guidance for personalized genetic medicines, establishing a regulatory pathway for individualized therapies targeting rare diseases, including gene editing and RNA-based treatments. The framework aims to facilitate the approval process by allowing approval based on a single well-controlled clinical investigation supported by confirmatory evidence, potentially accelerating the development of therapies like those used in the treatment of Baby KJ. While initially focused on rare genetic conditions, the guidance could extend to other individualized treatments, though it currently lacks detailed application instructions and does not address cancer indications. Several biotech companies, including Aurora Therapeutics, are leveraging this pathway to develop treatments for rare disease variants.

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C-SPAN addresses rumors that viral call was from Trump - USA Today

C-SPAN addresses rumors that viral call was from Trump - USA Today

C-SPAN clarified that a caller who sounded like Donald Trump and used the pseudonym "John Barron" was not the president, despite internet speculation. The caller, identified as a Virginia Republican, made comments critical of recent court decisions, prompting online jokes and skepticism. The use of the name "John Barron" by Trump in the 1980s as a pseudonym was noted, but C-SPAN confirmed the call was not from Trump himself. Trump is scheduled to deliver the State of the Union address on February 24.

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Who sits where at Trump's State of the Union address: A visual guide

Who sits where at Trump's State of the Union address: A visual guide

During President Trump's State of the Union address, Vice President JD Vance and House Speaker Mike Johnson are expected to sit behind him. In front, Cabinet members, Supreme Court justices, military leaders, and former Congress members will be seated, while other seats are unassigned and allocated on a first-come, first-served basis. The address typically includes around two dozen guests in the gallery, and the designated survivor, a Cabinet member ensuring continuity, is not present during the speech.

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After SCOTUS deals blow to Trump's tariffs, businesses wonder if refunds are coming - NPR

After SCOTUS deals blow to Trump's tariffs, businesses wonder if refunds are coming - NPR

The U.S. Supreme Court ruling nullified about half of President Trump's tariffs, raising uncertainty among businesses regarding potential refunds for over $100 billion in paid tariffs. Many small and large companies are yet to receive or process refunds, with some concerns over the administrative or legal complexities involved in claiming them. The case was sent back to a lower court for further resolution, leaving questions about how and when businesses will recover the funds.

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Kristi Noem “Completely Made Up” Story of ICE Deporting a Cannibal - Yahoo

Kristi Noem “Completely Made Up” Story of ICE Deporting a Cannibal - Yahoo

Kristi Noem falsely claimed that ICE deported a cannibal who tried to eat his own arms, a story later confirmed by multiple federal law enforcement officials to be completely fabricated. Noem’s account, which she shared in June, was unconfirmed and lacked evidence, with officials stating it never happened. The story was used by Noem to illustrate alleged dangers of deportations under Trump, but it has been widely debunked as a falsehood.

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Did President Trump call into C-SPAN under a fake name? - Poynter

Did President Trump call into C-SPAN under a fake name? - Poynter

A caller on C-SPAN who sounded like former President Donald Trump called in under the name “John Barron,” raising speculation that it was Trump himself, but C-SPAN clarified it was not the president. The incident sparked discussion about whether Trump would engage in such behavior, while he prepares for his upcoming State of the Union address amid mixed approval ratings. Additionally, the article covers recent developments in Warner Bros. Discovery’s potential sale, Conan O’Brien’s comments on a tragedy involving Rob Reiner’s family, the U.S. Olympic hockey victory, and notable reactions from sports legends.

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Dexter Sends Epstein Survivor to State of the Union in Her Place and Hosts Town Hall for ...

Dexter Sends Epstein Survivor to State of the Union in Her Place and Hosts Town Hall for ...

Congresswoman Maxine Dexter announced she is sending Epstein survivor Lisa Phillips to attend the State of the Union on her behalf to emphasize accountability and support for survivors. Dexter stated her intention to highlight that survivors will not be silenced and that the government should be held accountable. Phillips expressed gratitude and emphasized her purpose of advocating for those who lack a voice. Dexter also co-sponsored the Epstein Files Transparency Act to promote the release of relevant files and transparency.

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WATCH: Trump honors 'angel families' at the White House, repeats false claims about the ...

WATCH: Trump honors 'angel families' at the White House, repeats false claims about the ...

President Donald Trump hosted an "angel families" remembrance ceremony at the White House, honoring relatives of individuals killed by those illegally in the U.S., including Laken Riley, a woman murdered in 2024. The event also involved Trump signing a proclamation for "Angel Family Day" and was used to promote false claims that he only lost the 2020 election due to rigging, with no evidence supporting his allegations.

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Dr. Oz Warns Of 'Consequences' As People Choose Marijuana Over Alcohol, Citing ...

Dr. Oz Warns Of 'Consequences' As People Choose Marijuana Over Alcohol, Citing ...

Dr. Oz, head of CMS, warned that increased use of marijuana over alcohol could lead to negative consequences, citing concerns about high-dose hemp and CBD products. He emphasized potential risks associated with strong cannabis-derived products and highlighted ongoing efforts to include CBD coverage under federal health plans, as part of the Trump administration's cannabis rescheduling initiative. Oz has previously supported therapeutic cannabis use and advocated for policy changes to incorporate marijuana into healthcare options.

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Judge permanently blocks release of Jack Smith's report on Trump classified documents case

Judge permanently blocks release of Jack Smith's report on Trump classified documents case

A federal judge permanently blocked the Justice Department from releasing former special counsel Jack Smith's report on President Trump's handling of classified documents, citing concerns over protected discovery materials, statutory protections, and legal authority. The order prevents the release of the second volume of the report, which details the investigation into Trump's alleged misconduct, after efforts by Trump and his co-defendants to restrict its disclosure. The decision follows prior rulings and ongoing legal disputes regarding the report's public release.

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