With so much recent publicity surrounding gene therapy, it’s no surprise that the topic was a major focus of the…
Articles by Larry Luxner
Despite skyrocketing healthcare costs, President Trump is committed to protecting the 30 million or so Americans with rare diseases and…
A glance around the walls of Barry J. Byrne’s office reveals a lot about the pediatric cardiologist who runs the …
CureDuchenne has begun taking blood samples and skin biopsies to facilitate research on new treatments for Duchenne muscular…
About 700 people have registered to attend CureDuchenne’s Futures conference taking place Oct. 11-13 in suburban Los Angeles —…
Next month’s annual conference of the National Organization for Rare Disorders (NORD) in Washington, D.C., couldn’t come at a…
Many people are inspired by a son or brother with Duchenne muscular dystrophy (DMD). When it comes to Danielle Edwards,…
Developing gene therapies for rare diseases is one thing. Creating gene-edited “designer babies” is quite another. German legal expert Timo…
Imagine living your whole life with a painful disease so rare that only 25 others worldwide have what you have.
On a recent summer morning off the coast of Bat Yam, south of Tel Aviv, a dozen Israeli volunteers in…