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  • BioMarin's rare disease therapy shows no clinical benefit in Phase 3 test
    BioMarin’s enzyme replacement therapy for a rare genetic disorder called ENPP1 deficiency delivered mixed results in a late-stage study. Patients with the condition don't produce enough of the ENPP…
    Endpoints · 2mo ago↗ source
  • STAT+: 1 in 8 women drink during pregnancy. Experts dread the consequences
    More than 1 in 8 pregnant women report drinking. Doctors warn fetal alcohol disorders may be more common than many realize, perhaps more common than autism.
    STAT · 2mo ago↗ source
  • Opinion: Britain embraces a Massachusetts experiment on tobacco regulation — just as the state might roll it back
    Britain embraces a Massachusetts experiment on tobacco regulation — just as the commonwealth might roll it back.
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STAT · 2mo ago
↗ source
  • Opinion: I’m a heart transplant recipient. I’m haunted by ‘ghost approval’ for medication I need to survive
    “On paper, I have insurance coverage. In real life, I don’t have access. That’s what I call ‘ghost approval,’” writes patient advocate Payton Herres.
    STAT · 2mo ago↗ source
  • STAT+: Maryland state affordability board places a price cap on Ozempic
    Nine states have been pursuing the idea of prescription drug affordability boards. Maryland's board just set limits on the price of Ozempic.
    STAT · 2mo ago↗ source
  • STAT+: White House taps Amazon, GoodRx, and Mark Cuban to bolster TrumpRx
    Trump's drug discount platform adds 600 generic medications via major online pharmacies.
    STAT · 2mo ago↗ source
  • With no approved vaccine for Ebola outbreak, experts weigh testing a long shot
    With no approved vaccine for the fast-growing Ebola outbreak in the DRC, experts weigh testing one with unknown protection.
    STAT · 2mo ago↗ source
  • STAT+: Trump’s CMS to allow skimpier plans on ACA exchanges
    The new plans could leave patients on the hook for more of their health care costs.
    STAT · 2mo ago↗ source
  • Amgen stands by rare disease drug Tavneos amid Japan liver toxicity report
    The FDA wants it off the market. European regulators are investigating data-integrity concerns. And now, a pharma company selling the drug in Japan is warning doctors against writing new prescripti…
    Endpoints · 2mo ago↗ source
  • FDA's unreleased Covid vaccine deaths report is published by lawmaker
    An analysis launched by former FDA leaders to look into claims of pediatric deaths related to Covid-19 vaccines was published by a Republican lawmaker, months after the agency's self-imposed deadli…
    Endpoints · 2mo ago↗ source
  • STAT+: Why the WakeMed – Atrium Health hospital merger matters
    Hospital execs worked in secret on the deal for two years.
    STAT · 2mo ago↗ source
  • STAT+: AstraZeneca picks up FDA approval for hypertension drug
    The China question, FDA leadership shakeups, and other biotech news from The Readout
    STAT · 2mo ago↗ source
  • U.S. bans entry from Ebola-affected countries as American patient is identified
    In response to an Ebola outbreak, the U.S. has banned entry for noncitizens who have been in the Democratic Republic of the Congo, Uganda, or South Sudan recently.
    STAT · 2mo ago↗ source
  • Regeneron reaches $125M Parabilis deal for hard-to-catch drug targets
    As Regeneron’s cancer portfolio continues to suffer setbacks, the company is placing a bet in a different direction through a deal with Parabilis Medicines valued at as much as $2.2 billion. Parabi…
    Endpoints · 2mo ago↗ source
  • Design Therapeutics' Friedreich's ataxia data; Vincentage's obesity results from China
    Plus, news about Enhertu and Tecentriq. 💪 Design Therapeutics shares Phase 1/2 data in Friedreich's ataxia: The California biotech’s DT-216P2 given at 1 mg/kg led to an average 6.4-point improveme…
    Endpoints · 2mo ago↗ source
  • Merck's ADC sac-TMT gets its first global Phase 3 win ahead of schedule
    An experimental antibody-drug conjugate from Merck and China-based Kelun-Biotech succeeded in a global Phase 3 study for the first time, giving Merck a key pipeline victory as it approaches the pat…
    Endpoints · 2mo ago↗ source
  • STAT+: Supreme Court rejects challenge to Medicare drug price negotiation
    The rejection may make the pharma firms remaining lawsuits more difficult.
    STAT · 2mo ago↗ source
  • The Supreme Court won't take up drugmakers' IRA cases
    The Supreme Court says it won’t wade into the pharma industry’s yearslong legal battle over Medicare drug price negotiations. The justices declined to review multiple cases brought by AstraZeneca,…
    Endpoints · 2mo ago↗ source
  • STAT+: Pharmalittle: We’re reading about U.S. biotech’s China problem, a Regeneron flop, and much more
    The question of whether to partner with Chinese firms — or see them as rivals — is tearing biotech apart, pitting peers and partners against one another
    STAT · 2mo ago↗ source
  • Texas is opening up a detransition clinic
    Americans exposed to Ebola outbreak, Bill Cassidy's primary defeat, and more health news from Morning Rounds
    STAT · 2mo ago↗ source
  • Regeneron's Phase 3 skin cancer miss adds to mounting failures in LAG-3
    Regeneron Pharmaceuticals’ LAG-3 inhibitor called fianlimab has flunked a late-stage skin cancer trial, marking yet another blow for the drug class. The announcement follows a spate of recent clini…
    Endpoints · 2mo ago↗ source
  • FDA approves AstraZeneca’s baxdrostat for certain patients with hypertension
    One of the key products underpinning AstraZeneca’s goal of hitting $80 billion in revenue by 2030 has been approved by the FDA, the drugmaker said Monday. The blood ...
    Endpoints · 2mo ago↗ source
  • Opinion: ‘Patient autonomy’ has nothing to do with childhood vaccine policies
    Opinion: Vaccine policy it is not a matter of autonomy at all, because infants do not decline vaccination — their parents do.
    STAT · 2mo ago↗ source
  • STAT+: The China question is tearing biotech apart
    China's ascent is forcing U.S. biotech companies and investors to pick a side: Is it an ally or an existential threat?
    STAT · 2mo ago↗ source
  • Opinion: How to restore credibility to the CDC’s vaccine advisory committee
    Jesse Goodman, former director of CBER and former FDA chief scientist, calls for five critical changes to restore trust in the Advisory Committee on Immunization Practices.
    STAT · 2mo ago↗ source