leisure time -WSS

leisure time

author：hotspot 2025-09-27 12:56:43 - browse：87766

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： STAT Virtual Event: JPM Recap, Live!

WSS

Putin woos African leaders at a summit in Russia with promises of expanding trade and other ties

JPM 2024 Review: Hope for the XBI, deals from J&J, Merck, GSK

Mayo Clinic partners with supercomputer AI firm Cerebras at JPM

Adidas to release second batch of Yeezy sneakers after breakup with Ye

9 mass shootings over the weekend rock US cities, leaving 5 dead, 56 injured

JPM 2024: Biotech’s most prolific dealmaker sees a rosy 2024

focus

leisure time

hotspot

How one medical school became remarkably diverse

Arena BioWorks launches in Cambridge to speed drug discovery

Duchenne breakthrough therapy leaves behind pioneering families

Male doctors called my cancer incurable. A female doctor showed it wasn't

Hepatitis C has a cure — but many Americans still lack access to it

Male doctors called my cancer incurable. A female doctor showed it wasn't

Indiana Supreme Court upholds abortion ban, says state constitution gives only limited protections

Nonprofit, backed by Microsoft, to certify AI tools in health care

The cancer drug shortage isn’t new — and neither are the solutions

Trump team launching legal defense fund to help pay legal bills

explore

Doctors in states that ban abortion can still help patients

Biden has enormous polling lead in Democratic primary, despite challenge from RFK Jr.

Campaign finance charge dropped from case against Sam Bankman

Japan raises alarm over China's military, Russia ties and Taiwan tensions in new defense paper

Philippine ferry was overloaded when it flipped over, leaving 27 dead, official says

Doctors in states that ban abortion can still help patients

explore

entertainment

hotspot

Wikipedia