explore -WSS

explore

author：focus 2025-10-01 23:03:28 - browse：96844

Gene therapy helped Conner Curran, right, walk, but as the drug fades, his family is supporting scientific efforts to allow him and other boys get a second dose. -- Biotech coverage from STAT — Gene therapy helped Conner Curran (at right) walk, but as the drug fades, his family is supporting scientific efforts to allow him and other boys get a second dose. Courtesy Jess & Chris Curran

In the beginning, Conner Curran ran up the stairs.

It was a miraculous moment for him and his parents, Jess and Chris Curran. Just months before, contractors were in their New York home to install a motorized lift, while Jess tearfully explained that it wasn’t for a grandparent but for their 7-year-old son, who suffered from Duchenne muscular dystrophy and could barely haul himself up the steps.

Then Conner was accepted as the first patient in Pfizer’s Duchenne gene therapy trial. Three weeks later, he could bound to the second floor. Soon, he could walk the two-plus miles to Goldberg’s, a local bagel shop. “I couldn’t do that before,” he told NPR in 2020.

Unlock this article by subscribing to STAT+ and enjoy your first 30 days free!

GET STARTED Log In

Previous article： Anesthesiologist group: stop taking Ozempic before surgery
Next article： A clever new way to fix Medicaid and graduate medical education

WSS

HPV cancer vaccine study demonstrates efficacy

Teach medical students about patients with intellectual disabilities

Supreme Court wrestles with OxyContin maker's bankruptcy deal

In diabetes hearing, Sanders takes on a new foe: food makers

What causes an itch? Researchers may have solved the mystery

FDA approves world's first CRISPR

leisure time

explore

focus

Medical records are filled with copy

Study: New antibiotic targets gonorrhea as well as last existing drug

Cancer drug shortages should be causing more outrage

Diversify hospital leadership teams to protect BIPOC patient health

ChatGPT in medicine: STAT answers readers' burning questions

Pfizer plans to depart BIO

Moonlake's readout produced a cash windfall. Risks remain

In diabetes hearing, Sanders takes on a new foe: food makers

Psychedelics group wrestles with new pharma identity

White House calls on federal agencies to crack down on AI abuses

Wikipedia

Senate panel clears PBM, hopsital reforms

Removing probiotics from the NICU could threaten preemies' health

Supreme Court wrestles with OxyContin maker's bankruptcy deal

Spinal cord stimulation reduces pain in amputees, study finds

Pharma patents targeted by Biden in move to slash drug prices

Senate panel clears PBM, hopsital reforms

focus

focus

entertainment

entertainment