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  • Father spends $70 million on gene therapy trial for daughter with ultra-rare disease, faces FDA hurdles
    Matt Wilsey has spent a decade and about $70 million trying to develop a gene therapy for his 15-year-old daughter Grace, who suffers from a fatal ultra-rare disease. His personal quest has turned…
    STAT · 1mo agoNegative↗ source
  • Michigan sees nearly 1,000 cyclospora cases, the state's largest outbreak and among the nation's biggest
    Nearly a thousand residents of Michigan have been diagnosed with a cyclospora infection that causes prolonged watery diarrhea, making it the biggest outbreak in the state's history and one of the l…
    STAT · 1mo agoNeutral↗ source
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ARPA-H launches $160 million THRIVE program to fund seven labs developing custom gene-editing therapies for rare diseases
ARPA-H, the U.S. Advanced Research Projects Agency for Health, announced a new initiative called THRIVE with up to $160 million in funding. The program will support seven research teams over five…
STAT · 1mo agoPositive↗ source
  • Prime Secures Arbitration Victory Over Beam, Paving Way for Clinical Trials
    Prime, a biotech firm developing a gene‑therapy candidate, won an arbitration case against Beam Therapeutics. The ruling removes a legal hurdle and allows Prime to advance its program into clinical…
    STAT · 1mo agoRegulatoryPositive↗ source
  • AstraZeneca/Ionis heart-failure drug Wainua fails pivotal trial as White House reviews FDA commissioner candidates
    The White House is reviewing a shortlist of three candidates for the FDA commissioner position, according to STAT. The list includes White House adviser Heidi Overton, oncologist and health‑system…
    STAT · 1mo agoNegative readoutNegative↗ source
  • Father invests $70 million in gene therapy quest for daughter with ultra‑rare NGLY1 deficiency, faces FDA scrutiny
    Matt Wilsey has spent a decade and roughly $70 million trying to develop a gene therapy for his daughter Grace, who suffers from NGLY1 deficiency, an ultra‑rare genetic disorder. NGLY1 deficiency…
    STAT · 1mo agoRegulatoryPositive↗ source
  • Northwest Biotherapeutics' brain cancer therapy faces 931-day delay in UK approval
    Northwest Biotherapeutics, a public biotech developing a brain cancer treatment, filed a marketing application with the UK Medicines and Healthcare products Regulatory Agency (MHRA) in late Decembe…
    STAT · 1mo agoRegulatoryNegative↗ source
  • New OpenEvidence study shows it outperforms general-purpose LLMs in health-tech research
    A recent study funded by OpenEvidence compared its platform to general-purpose large language models (LLMs) in health technology research and concluded that OpenEvidence delivered more accurate and…
    STAT · 1mo agoPositive↗ source
  • White House reviews three candidates to lead the FDA
    STAT+ reports that the White House is currently evaluating three leading candidates to become the next head of the Food and Drug Administration. The review process is part of the administration's e…
    STAT · 1mo agoRegulatoryNeutral↗ source
  • Doctors urge safe medical cannabis use as more seniors turn to marijuana
    Older adults are becoming the fastest‑growing segment of medical cannabis patients, according to the author, an addiction specialist at Massachusetts General Hospital. He recounts a recent inciden…
    STAT · 1mo agoNeutral↗ source
  • Experts question commercial motives behind labeling obesity as a disease
    Max Moser, a psychology researcher at University College London, argues that calling obesity a disease gives comfort to some patients but also creates a commercial dynamic for drugmakers. Companie…
    STAT · 1mo agoNegative↗ source
  • Podcast discusses health insurance costs, whistleblower case, and potential FDA commissioner picks
    The Readout LOUD podcast episode explores why employer-provided family health insurance can be as expensive as a new car. The hosts interview a whistleblower who challenged an insurance contractor…
    STAT · 1mo agoRegulatoryNeutral↗ source
  • Health officials discuss screen-time harms at Great American State Fair's MAHA Monday
    The Great American State Fair in Washington concluded its two‑week run with a second “MAHA Monday,” a program highlighting the Make America Healthy Again initiative. STAT reporters Alex Hogan and…
    STAT · 1mo agoNeutral↗ source
  • White House conducts final review of three candidates for FDA commissioner
    The White House is completing its final review of candidates to become the next Food and Drug Administration commissioner. The shortlist features Heidi Overton, a current White House adviser; Jeff…
    STAT · 1mo agoRegulatoryNeutral↗ source
  • U.S. officials urge pharma companies to increase domestic production of essential generic drugs
    At a private gathering in the Eisenhower Executive Office Building, senior officials from the Trump administration met with pharmaceutical leaders. Secretary of State Marco Rubio, Health Secretary…
    STAT · 1mo agoRegulatoryNeutral↗ source
  • Lung transplants boost survival for select stage 4 lung cancer patients, study finds
    Northwestern Medicine surgeons and oncologists performed double lung transplants on a small group of stage 4 lung cancer patients whose disease was confined to the lungs. The JAMA study compared 1…
    STAT · 1mo agoPositive readoutPositive↗ source
  • Europe to Speed Review of RevMed Drug as Concerns Rise Over Kailera GLP-1 Pill and FDA Clears Vera Therapy
    European regulators said they will move more quickly to assess RevMed's investigational drug, aiming to shorten the evaluation timeline. Separate reports highlighted that recent data from Kailera'…
    STAT · 1mo agoRegulatoryNeutral↗ source
  • Prime Medicine wins arbitration over gene-editing dispute with Beam, clearing path for AATD trial
    Prime Medicine announced it had prevailed in an arbitration against Beam Therapeutics, ending a dispute over gene-editing technology. The arbitrator determined that Prime’s work on a gene-editing…
    STAT · 1mo agoRegulatoryPositive↗ source
  • FDA approves Vera Therapeutics' Trutakna for IgA nephropathy, pricing set at $425,000 per year
    The U.S. Food and Drug Administration has granted approval to Trutakna, a new therapy from Vera Therapeutics, for the treatment of IgA nephropathy. IgA nephropathy is a kidney disorder where immun…
    STAT · 1mo agoApprovalPositive↗ source
  • ACA insurers seek median 14% premium hike for 2027 as enrollment falls
    Insurers filing with state regulators for 2027 ACA plans are asking for a median premium increase of 14%, according to an analysis of filings in 16 states and the District of Columbia. This would…
    STAT · 1mo agoRegulatoryNegative↗ source
  • FDA targets online ketamine sellers as health premiums surge, and children 2+ can now obtain CGMs without prescription
    The Food and Drug Administration is intensifying its enforcement against online retailers that sell ketamine, a controlled substance, citing concerns about unregulated distribution and public safet…
    STAT · 1mo agoRegulatoryNeutral↗ source
  • AI licensure debate overlooks physician responsibility for patient outcomes
    The authors argue that discussions about licensing artificial intelligence tools in medicine miss the core purpose of licensure, which is accountability for clinical results. They illustrate a scen…
    STAT · 1mo agoNeutral↗ source
  • Trump administration seeks lasting overhaul of federal science policy after court setbacks
    The White House has introduced a proposal to cement its authority over federal research contracts, allowing the executive branch to direct research, end grants, and control external scientific revi…
    STAT · 1mo agoRegulatoryNegative↗ source
  • AI chatbots could provide companionship for people with dementia, but their role raises complex questions
    Jason Karlawish, a professor at the University of Pennsylvania, writes an opinion piece exploring how artificial-intelligence chatbots might be used in dementia care. He recounts the story of Doug…
    STAT · 1mo agoNeutral↗ source
  • Mass General Brigham nurses begin largest strike in Massachusetts over pay and benefits
    Thousands of nurses at Brigham and Women’s Hospital walked off the job on Wednesday, marking the first strike at the Harvard‑affiliated facility and the largest nurses walkout in the state’s histor…
    STAT · 1mo agoNegative↗ source