In a first, nonprofit wins approval for rare disease therapy

Taking stock of the drug industry, from the lab to the medicine chest

December 10, 2025 Check out STAT+

Sponsored By

By Ed Silverman Pharmalot Columnist, Senior Writer

Agnese Morganti for STAT STAT+ | In a first, a nonprofit wins FDA approval for rare disease gene therapy The FDA approved a new gene therapy for a rare immune disorder, the maker of which is notably not a drug company, but an Italian charity.  By Andrew Joseph Myocarditis is a rare but real Covid vaccine side effect. A new study sheds light on what might cause it Study points to source of "very rare" cases of myocarditis and offers potential solutions. Myocarditis risk remains far higher from Covid infection than the vaccine. By Jonathan Wosen Opinion: The revolution in dementia care is just beginning Jason Karlawish on how diagnostics and treatment for Alzheimer's and other causes of dementia are changing completely. By Torie Bosch Justin Sullivan/Getty Images STAT+ | Major health insurers to skip J.P. Morgan conference, again High costs, quiet-period rules, investor skepticism, and lingering safety concerns are pushing major insurers to once again skip JPM's flagship event. By Tara Bannow and Bob Herman

More around STAT

Check out more exclusive coverage with a STAT+ subscription Read premium in-depth biotech, pharma, policy, and life science coverage and analysis with all of our STAT+ articles.

---

Enjoying Pharmalot? Tell us about your experience Continue reading the latest health & science news with the STAT app Download on the App Store or get it on Google Play

STAT, 1 Exchange Place, Boston, MA ©2025, All Rights Reserved. I no longer wish to receive STAT emails

Update Newsletter Preferences     •    Contact Us     •    Advertise with Us     •    View in Browser