Core Insights - Ultragenyx Pharmaceuticals (RARE) has received FDA Breakthrough Therapy designation for its investigational therapy GTX-102 (apazunersen) aimed at treating Angelman syndrome (AS) [1][5]. FDA Breakthrough Therapy Designation - The Breakthrough Therapy designation accelerates the development and review of drugs for serious conditions, granted when early clinical evidence indicates significant improvement over existing treatments [2]. - Drugs with this designation benefit from enhanced guidance and support from senior FDA officials [2]. Clinical Study Results - GTX-102 demonstrated rapid and lasting improvements in AS patients during a phase I/II study lasting up to three years, involving 74 patients aged 4 to 17 with complete maternal UBE3A gene deletion [5][6]. - Participants showed consistent developmental progress and ongoing improvements across multiple symptom areas during treatment [6]. Current and Future Studies - Ultragenyx is currently enrolling patients for a phase III Aspire study and plans to initiate a phase II/III Aurora study to evaluate GTX-102 for a broader range of AS genotypes [8]. - The Aurora study is expected to start in the second half of 2025 [8]. Market Context - AS is a rare neurogenetic disorder affecting approximately 60,000 individuals in accessible markets, leading to severe developmental challenges with no approved therapies currently available [7]. Other Clinical Programs - Ultragenyx is developing other gene therapy candidates, including UX143 for osteogenesis imperfecta, which received Breakthrough Therapy designation in October 2024 [9]. - The company is also evaluating UX701 for Wilson disease and has achieved significant results in the phase III GlucoGene study for glycogen storage disease type Ia [10]. - A regulatory application for UX111, an AAV gene therapy for Sanfilippo syndrome type A, is under Priority Review, with a decision expected on August 18, 2025 [11].

[Music] You have a war to fight. A small sentence, just five words, but it changed everything. I was lying in a hospital bed when the doctor examined my test results and said those words to me.My 8-year-old self was baffled. Seriously, me fighting a war. What war.At eight years old, how was I supposed to fight something as big as a war. Wars were things I read about in history books, saw on the news, or heard about in the stories my grandfather told me. But I was just a kid.It turns out that the war I had t ...

Russia summoned Azerbaijan’s ambassador after police in Baku raided the office of a Russian state-funded media outlet as ties continue deteriorating between the former Soviet neighbors https://t.co/miLG6RBsBD ...

We are now joined by U.S. Treasury Secretary Scott Betts. And just as the Senate has begun voting on what's known as the big, beautiful bill. You look at the latest version of the tax bill.The big question that remains is the GOP holdouts. What kind of deal can be cut with those holdouts. And what are you and the president prepared to offer for support.Well, I'm confident that the bill is going to progress, as is over the next few hours, and it'll be on the president's desk to sign on July 4th. So the Senat ...

A debtor nation has negative net investment after recording all of the financial transactions it has completed worldwide. https://t.co/Fo5DltJYDq ...

Just as the Senate has begun voting on what's known as the big, beautiful bill. You look at the latest version of the tax bill. The big question that remains is the GOP holdouts.What kind of deal can be cut with those holdouts. And what are you and the president prepared to offer for support. Well, I'm confident that the bill is going to progress, as is over the next few hours, and it'll be on the president's desk to sign on July 4th.So the Senate Senate will vote, pass it over to the House. We've seen incr ...

Group 1 - The Pragmatic Investor focuses on global macro, international equities, commodities, tech, and cryptocurrencies, aiming to guide investors of all levels [1] - The platform offers features such as a portfolio, weekly market updates, actionable trades, technical analysis, and a chat room for investor engagement [1] - James Foord, an economist with a decade of experience in global market analysis, leads The Pragmatic Investor, emphasizing the creation of diversified portfolios to preserve and grow wealth [1]

Let's turn now to Washington where later this morning the Senate is expected to begin what's known as voterama only in Washington DC on President Trump's sweeping domestic policy package. It comes after late on Saturday the Republican le Senate advanced the package following a dramatic drawn out process that spanned hours bringing it one step closer to passage. It narrowly advanced after Majority Leader John Thoon and Vice President JD Vance struck a deal with the holdouts.Trump also stayed in Washington ov ...

Kazakhstan plans national cryptocurrency reserve using seized assets, state-mined coins https://t.co/z394zlHjZH ...

Wilmington, DE, June 30, 2025 (GLOBE NEWSWIRE) -- Aimfinity Investment Corp. I (the “AIMA”) (Nasdaq: AIMTF), a special purpose acquisition company incorporated as a Cayman Islands exempted company, today announced that, in order to extend the date by which the Company mush complete its initial business combination from June 28, 2025 to July 28, 2025, on JUne 28, 2025, I-Fa Chang, manager of the sponsor of the Company, has deposited into its trust account (the “Trust Account”) an aggregate of $55,823.8, or f ...