explore -WSS

explore

author：focus 2025-10-01 17:18:51 - browse：7

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： BioMarin wins approval for gene therapy to treat hemophilia A
Next article： United colonoscopy coverage change 'may cost lives,' doctors say

focus

WSS

Apple is now the first public company to be valued at $3 trillion

Wildfire smoke exposes gaps in outdoor worker protections

Idaho abortion case may prompt more restrictions in other states

Sports medicine is finally prioritizing gender equality

Health and fitness trackers are measuring the wrong things

United colonoscopy coverage change 'may cost lives,' doctors say

hotspot

explore

explore

FDA approval of Sarepta Duchenne genetic therapy gives me hope

Doudna institute plans to 'cure hundreds of diseases' with CRISPR

Apple is now the first public company to be valued at $3 trillion

Novavax promises a turnaround & Lilly roils the obesity market

Alkermes shareholders re

Health and fitness trackers are measuring the wrong things

Virginia high school admissions case could be legal follow

HHS names first

Jerome Adams on the need for new antivirals for Covid

Idaho abortion case may prompt more restrictions in other states

hotspot

JPM 2024: FogPharma’s CEO unveils ‘contrarian’ plan in oncology

Activists slam private health insurers over coverage restrictions

How one medical school became remarkably diverse

Why liver experts renamed NASH to MASH, and NAFLD to MASLD

Why liver experts renamed NASH to MASH, and NAFLD to MASLD

Trump demands the U.S. pay no more for drugs than other countries … again

focus

Wikipedia

hotspot

explore