Metformin, the first line drug for Type 2 diabetes, has been used for 60 years without a clearcut mechanism of action. It turns out it's not just reducing glucose output from the liver. Metformin also works via the brain www.science.org/doi/10.1126/...

“This is not a trivial decision, and the emotional rollercoaster for families is unfair and vicious.”
For many Duchenne families, halt to gene therapy is heartbreak upon heartbreak www.statnews.com/2025/07/24/d...

What was achieved for Baby KJ in 6 months was inconceivable (Timeline below). The new Center will try to go even faster for lifesaving treatment of babies with rare diseases @ucsfmedicine.bsky.social @ucberkeleyofficial.bsky.social @urnov.bsky.social

Playing video games or finding better ways to read 3D echocardiograms? Learn how using VR, XR and 3DE can help provide more accurate diagnoses. #CardioSky #VirtualReality #PedsCardiology

U.S. hospitals lost ~30% of pediatric inpatient units from 2008 to 2022, “dramatically impacting rural areas and leaving millions of children without direct access to pediatric inpatient care,” says Dr. Emily Bucholz, co-author of a @jama.com Pediatrics study.

New @nature.com
An ingenious way to track DNA replication patterns at the single-cell level that leads to cell heterogeneity and daughter cells that can potentially give rise to cancer
www.nature.com/articles/s41...

The RFK Jr. version of health, where health creates health and healthy people don't get sick and the thing that keeps you healthy is /living the right way/ is very seductive to a lot of people. It also places the burden of illness back on the ill, which decreases the value of human life.

Today "a milestone in the evolution of personalized therapies for rare & ultra-rare inborn errors of metabolism"
—the 1st human to undergo custom genome editing
—from decades of NIH funded research
www.nejm.org/doi/full/10....
@nejm.org
www.nejm.org/doi/full/10....
www.nytimes.com/2025/05/15/h...

The deeper meaning behind "almost no healthy children die of the measles" is that, when children do die of the measles, it's only ever because they were already ill. And the deeper meaning behind that is "well, we can't save 'em all." And that's just...17th century thinking.

The new Neil Halloran documentary on Smallpox has completely changed how I see the RFK Jr. version of the world. Neil (softly) argues that realizing that disease isn't the fault of the sick /or/ the fault of the Gods ushered in a new future where we felt the authority to care for each other.