Once-divisive rare disease treatments are showing startling promise

A mother's persistence helps revive an all-but-abandoned drug class and could aid her son and many others with neuromuscular conditions. ͏  ͏  ͏  ͏  ͏  ͏  ͏  ͏  ͏  ͏  ͏  ͏  ͏  ͏  ͏  ͏  ͏  ͏  ͏  ͏  ͏  ͏  ͏  ͏  ͏  ͏  ͏  ͏  ͏  ͏  ͏  ͏  ͏  ͏  ͏  ͏  ͏  ͏  ͏  ͏  ͏  ͏  ͏  ͏  ͏  ͏  ͏  ͏  ͏  ͏  ͏  ͏  ͏  ͏  ͏  ͏  ͏  ͏  ͏  ͏  ͏  ͏  ͏  ͏  ͏  ͏  ͏  ͏  ͏  ͏  ͏  ͏  ͏  ͏  ͏  ͏  ͏  ͏  ͏  ͏  ͏  ͏  ͏  ͏  ͏  ͏  ͏  ͏  ͏  ͏  ͏  ͏  ͏  ͏  ͏  ͏  ͏  ͏  ͏  ͏  ͏  ͏  ͏  ͏  ͏  ͏  ͏  ͏  ͏  ͏  ͏  ͏  ͏  ͏  ͏  ͏  ͏  ͏  ͏  ͏ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­  

April 8, 2026 Check out STAT+

Sponsored By

Get your team access to STAT+ and save more than $170/person. A STAT+ subscription offers unlimited access to award-winning journalism, exclusive data tools, and in-depth analysis. Check out this limited-time offer. Thanks for reading!

Sandy Huffaker for STAT STAT+ | A decade ago, these drugs tore apart the FDA. Today, they might be some patients’ best hope An exon-skipping drug delivered striking results, but the Duchenne muscular dystrophy field has been burned before and some fear history could repeat. By Jason Mast STAT+ | Everyone agrees AI scribes are increasing health care costs. No one agrees what to do about it It's unclear how the health care industry will handle technology-driven inflation, especially in the face of political pressure to lower health care costs. By Brittany Trang STAT+ | 23andMe finds genetic changes appear to help predict response to GLP-1 drugs for weight loss New research by the 23andMe Research Institute suggests genetic changes appear to help predict whether patients will lose substantial weight on GLP-1 drugs. By Matthew Herper Opinion: How sports betting is hooking some young men Isaac Rose-Berman on the rise of sports gambling as public health threat — and what to do about it. By Torie Bosch STAT+ | Steve Ubl to step down as CEO of PhRMA The powerful pharmaceutical industry lobbying group PhRMA has faced off with both Republicans and Democrats over efforts to rein in drug prices. By John Wilkerson Smart contact lens monitors eye pressure and delivers glaucoma drugs in early tests Device monitors ocular pressure without need for electronics or a battery, and delivers pulses of glaucoma drug as needed.  By Annalisa Merelli David Ryder/Getty Images Opinion: What American hospitals can learn from India about waste When these medical students visited hospitals in India, they learned that medicine does not have to be so bad for the environment. By Srinidhi Polkampally and Bhav Jain

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 Daily Recap? 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 ©2026, All Rights Reserved. I no longer wish to receive STAT emails

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