
FDA approves first CIRM‑funded gene‑editing therapy for rare immune disorder
The FDA has granted approval to KRESLADI, a gene‑editing therapy from Rocket Pharmaceuticals that treats severe leukocyte adhesion deficiency‑I in children without a matching bone‑marrow donor. It is the first product directly funded by California’s Institute for Regenerative Medicine (CIRM), highlighting the agency’s impact on translational research.
Also developing:
By the numbers: Syneron Bio raises $150M Series B

Advocates are urging the public to sign a petition calling for the repeal of the 1986 liability shield that protects medical product manufacturers from lawsuits. The campaign highlights two pending bills—S.3853 introduced by Rand Paul and H.R.4668 by Rep. Gosar—aimed at restoring accountability and consumer safety. Organizers argue the upcoming midterm elections create a unique political window, with growing public support and a new HHS leadership willing to challenge the status quo. They contend that without legislative change, manufacturers lack incentives to produce safer products.

A real‑world study of 20,571 California patients with aortic stenosis found that echocardiographic surveillance aligned with 2020 ACC/AHA guidelines markedly improves outcomes. Guideline‑concordant monitoring occurred in 74% of mild cases but fell below 50% for severe disease, the group at...

Reform‑oriented politicians are increasingly at odds over health‑care strategies, with some pushing universal coverage and price controls while others favor market‑based solutions. The debate has intensified as think tanks and advocacy groups publish competing policy briefs, reflecting broader shifts toward...

Repeated head impacts in contact sports have been linked to lasting damage of the blood‑brain barrier (BBB), a finding that may underlie chronic traumatic encephalopathy (CTE). Researchers scanned 47 retired athletes using an MRI contrast agent that only enters brain...

Researchers using a machine‑learning algorithm estimate that U.S. COVID‑19 deaths in 2020‑2021 were 150,000‑160,000 higher than official CDC counts, raising the total to nearly one million. The study examined 5.7 million adult death records, flagging likely COVID deaths that occurred outside...

In the latest Elevate Podcast, Hospice News interviews Dr. Brian Haas, national medical director of Ascend Hospice and CEO of Wellspring Healthcare. Haas explains how artificial intelligence can both help and hinder clinical documentation in hospice and palliative care. He...

The latest JACC statistics issue reveals that the long‑standing decline in cardiovascular mortality has stalled, while total spending on cardiovascular care has more than tripled since 2000. Analysis of privately insured working‑age adults shows inflation‑adjusted healthcare expenditures rose from $4,813...

The Trump administration’s 2027 Notice of Benefit & Payment Parameters (NBPP) would allow ACA Marketplace insurers to sell “non‑network” plans that set a fixed payment amount for services instead of contracting with providers. Under the proposal, patients would be responsible...

OCHIN and Community Care Cooperative (C3) have launched a joint accountable care organization (ACO) offering tailored for federally qualified health centers (FQHCs). OCHIN will supply its Epic EHR platform and analytics, while C3 brings a suite of Medicare ACO services,...

The FDA released a draft guidance that details how biopharma firms can validate non‑animal approaches for early‑phase toxicology and safety studies. The document encourages the use of in‑vitro assays, computational modeling, and other modern methods to replace traditional animal testing....

The Iranian‑linked Handala Team launched a wiper attack on Stryker Corporation on March 11, destroying the Lifepak cardiac monitor network that links ambulances to hospitals. The outage halted real‑time ECG transmission in Maryland, jeopardizing STEMI patients and exposing the shared vulnerability...

Governor Ron DeSantis' attempt to roll back Florida vaccine mandates has encountered federal legal and regulatory barriers, limiting the state's ability to alter existing health program requirements. The push, framed as a "medical freedom" initiative, now faces challenges that could...

Harvard-affiliated researchers used AI to evaluate routine CT scans and discovered that a healthy thymus in adults predicts markedly lower mortality, cardiovascular death, and lung cancer risk. The studies, covering over 25,000 participants from a lung‑cancer screening trial and the...

Azalea Therapeutics, a spinout from Jennifer Doudna’s lab, reported in Nature that its in vivo CAR‑T approach can generate functional CAR‑T cells directly within mice and eradicate both solid and hematologic tumors. The technique uses infused gene‑editing particles that precisely...
Is "textbook performance" enough for medical AI? The first independent stress test of ChatGPT Health is out in @NatureMedicine. The Good: Near-perfect triage for textbook stroke and anaphylaxis. The Bad: A 51.6% under-triage rate for true emergencies. But there’s a massive elephant in the...

The post argues that surgeon awareness creates interest, which leads to adoption and ultimately reshapes orthopedic markets. OTW positions itself as a catalyst by showcasing research to the most influential spine surgeons. It invites researchers to submit their work through...

JenaValve received FDA clearance for its Trilogy Transcatheter Heart Valve System, marking the first TAVR device expressly approved to treat symptomatic, severe aortic regurgitation (AR). The approval follows the ALIGN‑AR trial, which demonstrated the valve’s safety and efficacy in a...
At DevSparks 2026, Partex.AI unveiled a sovereign healthcare foundation model that combines federated learning, knowledge graphs, and agentic AI to analyze fragmented medical data without moving patient records. The architecture keeps data on‑site, sharing only encrypted model updates, while knowledge...

Mass General Brigham Health Plan partnered with mPulse to launch an interactive wellness hub for commercial, Medicare Advantage, Medicaid and dual‑eligible members. The platform uses predictive analytics to deliver personalized nutrition, activity, sleep and stress‑management tools through audio, visual and...

Valbenazine (Ingrezza®), an oral selective VMAT2 inhibitor from Neurocrine Biosciences, received FDA approval for treating tardive dyskinesia and Huntington’s disease‑associated chorea. In the Phase 3 KINECT‑3 trial, a once‑daily 80 mg dose produced a statistically significant reduction in AIMS dyskinesia scores after...

UniQure’s experimental gene‑therapy for Huntington’s disease, which previously reported a 75% slowdown in disease progression, has received a third consecutive rejection from the FDA. The trial’s lead investigator, Ed Wild of University College London, praised the early data but warned...

Aetna has introduced a black‑box algorithm that automatically downcodes physician claims, lowering payments for high‑severity ER visits without chart review. The system reclassifies Level 5 services to Level 4, forcing doctors to submit appeals to the same insurer that made the reduction....
Alabama lawmakers are advancing HB 269 and its companion HB 400 to overhaul ambulance reimbursement rates and allow "treat‑in‑place" billing, aiming to keep rural EMS providers financially viable. The measures respond to an "ambulance desert" landscape where response times can exceed an...

Basecamp Research, an AI‑focused biotech startup backed by Microsoft and Nvidia, announced a trillion‑gene sequencing initiative. The company aims to collect genetic sequences for over a trillion proteins within the next two years. Leveraging high‑performance cloud computing and advanced generative‑AI...
If you are vibe-coding apps for the healthcare sector I beg you - pay a deeply experienced technologist to audit it before anyone uses it. hipaa laws are no joke. I’ve personally been through the compliance audits, defending our encryption...

A Tennessee bill that would have treated abortion as criminal homicide failed to advance, not because of public opposition but due to procedural inaction. The proposal, which could have exposed women to the death penalty, highlights how even the most...

Dr. Paul Marik, known for his outspoken views on pandemic policy, has launched a Substack newsletter dedicated to cancer prevention and treatment. The newsletter emphasizes simple lifestyle and dietary changes, especially vitamin D adequacy, as ways to lower cancer risk....
In early March 2026 four NHS trusts in Somerset and Dorset signed a £222 million federated contract with US‑based Epic to replace a patchwork of legacy electronic patient record (EPR) and patient‑administration systems. The deal unifies Epic’s platform across Somerset NHS...

Ionis Therapeutics announced that the primary completion date for its Phase 1/2a prion disease trial has been pushed to February 2027, extending the study timeline by more than two years. In parallel, Arvinas reported new biomarker data from its Parkinson’s disease program,...
On November 20, 2020 Johnson & Johnson’s Verb division unveiled OTTAVA, a six‑armed robotic platform designed for minimally invasive surgery. The system combines modular hardware with AI‑driven control to perform simultaneous tasks, promising greater precision and efficiency. Early trials indicate up to...

From a clinical perspective, patients are not asking for abstract years. They are asking for time with family, time in full cognition, time in independence, and time living on their own terms. That is why geroscience should be judged not...
CBER changes its standard operating procedures to open the door to RMAT designations for therapies on clinical hold - https://t.co/bZmhga8xxC

Incoming ACC President Roxana Mehran discusses the upcoming 2026 ACC meeting in New Orleans on the Rox Heart Radio podcast. She outlines her vision for the organization, emphasizing data‑driven leadership, patient‑centered outcomes, and broader member engagement. The episode highlights strategic...

Sharp take here from healthcare VC Sam Toole on the future of EHRs in an AI world. Do they become systems of record, and entrepreneurs can build systems of action that sit on top? Link to read the full piece...

What if we could engineer T cells in the body, making CAR T vs cancer, autoimmune diseases, and other applications far more practical, much less expensive? A step forward today via genome editing T cells https://t.co/FjgQA8NUlu https://t.co/XXk43WL1ac https://t.co/rpC7eCAlJH
Structure Therapeutics reported a 16.3% weight loss after 44 weeks in a Phase 2 trial of its oral GLP‑1 pill, positioning it as a competitive alternative to Eli Lilly’s and Novo Nordisk’s candidates. Rhythm Pharmaceuticals disclosed that its obesity drug Imcivree failed in...
A randomized clinical trial of repeated doses of psilocybin for the treatment of obsessive–compulsive disorder 73.3% were responders (⩾35% reduction in YBOCS scores), with 40% in remission. These effects diminished but remained substantial at 6 months. https://t.co/tp2SPIKDrB
For anyone considering immigrating to Uruguay here's a great overview of how the medical system works and how immigrants can sign up for either public or private healthcare. https://t.co/TZTE9AwEkv

Crossbow Therapeutics, founded by biotech veteran Briggs Morrison, announced a $77 million Series B round to accelerate its off‑the‑shelf T‑cell engager platform. The funding, led by a mix of venture capital and strategic investors, will support the advancement of three preclinical candidates...
RBC: Revolution, Xenon, Arrowhead among top #biotech takeover targets https://t.co/h1toH9szrU by @realJacobBell $ARWR $XENE $RVMD $DYNE
I now realize that the "solution" to the rising cost of healthcare will not be to ration healthcare. It will be to kill the most expensive patients.

Aspen Neuroscience reported encouraging early data from a small autologous cell therapy trial for Parkinson's disease. The treatment, derived from each patient's own skin cells and reprogrammed into dopaminergic neurons, was safely implanted in twelve participants, showing graft survival and...

Potential of mitochondrial transfer to prevent or treat Parkinson' s disease, in mouse and monkey models @CellCellPress https://t.co/c0oqfagddX https://t.co/KyBc1zQttc
Does it stack the deck in favor of the standard antidepressants to pull data from placebo-controlled trials for one condition (the psychedelic) and compare it with single condition data for the standard antidepressants? Also...

Tommy Macnally, now a recovery worker at Antidote, recounts his eight‑year descent into chemsex—a subculture of drug‑enhanced sex among GBMSM—followed by a painful but successful sobriety journey. National data show drug‑poisoning deaths are 2.8 times higher for LGBTQ+ people, with London...

80% of healthcare practices that invest in AI automation fail to see ROI. Not because the tools don't work, but because there was no digital strategy. Don't be the 80% See more & JoinUs https://t.co/05FgGDavR7 #healthcare #ai #businessautomation #smallbusiness #medical #doctor...
How to get rich on out-of-network medical bills -- and how to get angry reading about it. By @TaraBannow https://t.co/fbmFyZhcic
A recent Indian study of 91 patients exposed to alternative medicines found that 39.6% developed acute‑on‑chronic liver failure (ACLF), with a 38.9% mortality rate among those cases. Heavy‑metal contamination exceeded WHO limits in many products, and 27.7% contained undeclared pharmaceutical...

The UK’s Medicines and Healthcare products Regulatory Agency (MHRA) and the National Institute for Health and Care Excellence (NICE) will begin delivering aligned guidance in June, enabling simultaneous licensing and reimbursement decisions for new medicines. Twenty‑seven companies have signed up...

Sanofi’s oral glucosylceramide synthase inhibitor venglustat has earned U.S. FDA breakthrough therapy designation for treating the neurological aspects of type 3 Gaucher disease. The designation follows the Phase III LEAP2MONO trial, which showed significant improvements in motor and cognitive scores compared with...