With so much recent publicity surrounding gene therapy, it’s no surprise that the topic was a major focus of the recent 2019 NORD Rare Diseases & Orphan Products Breakthrough ... Read more
Despite skyrocketing healthcare costs, President Trump is committed to protecting the 30 million or so Americans with rare diseases and ensuring timely, affordable access to lifesaving treatments, the nation’s ... Read more
A glance around the walls of Barry J. Byrne’s office reveals a lot about the pediatric cardiologist who runs the Powell Gene Therapy Center at University of Florida (UF). In ... Read more
CureDuchenne has begun taking blood samples and skin biopsies to facilitate research on new treatments for Duchenne muscular dystrophy. The CureDuchenne Biobank is a partnership involving neurologist Tahseen Mozaffar, ... Read more
About 700 people have registered to attend CureDuchenne’s Futures conference taking place Oct. 11-13 in suburban Los Angeles — more than six times the number of participants at the ... Read more
Next month’s annual conference of the National Organization for Rare Disorders (NORD) in Washington, D.C., couldn’t come at a better time, says Marshall Summar, MD, chairman of NORD’s board ... Read more
Many people are inspired by a son or brother with Duchenne muscular dystrophy (DMD). When it comes to Danielle Edwards, she’s literally made the disease her career. Edwards, 26, ... Read more
Developing gene therapies for rare diseases is one thing. Creating gene-edited “designer babies” is quite another. German legal expert Timo Minssen outlined the potentially explosive ethical landmines surrounding such ... Read more
Imagine living your whole life with a painful disease so rare that only 25 others worldwide have what you have. And that you’re one of just six such people ... Read more
On a recent summer morning off the coast of Bat Yam, south of Tel Aviv, a dozen Israeli volunteers in wetsuits splashed in the sparkling blue waters of the ... Read more
