Adam's Blogroll: click through to the author's blog
The news this week presented at a major vision conference and published in The New England Journal of Medicine about gene therapy to treat childhood blindness paints an unnecessarily grim picture. Because I wrote a book about it and know affected families, … Continue reading
The post Gene Therapy for Blindness Works! appeared first on PLOS Blogs Network.
Levi Collazo, a biology major at Southwestern Oklahoma State University, has cystic fibrosis (CF). “In the first photo, I was making fun of my own weight. I’ve always used humor as a defense mechanism. I weighed 110 pounds. I’m 5’10″. … Continue reading
The post New Miracle Drugs: What Would You Pay? appeared first on PLOS Blogs Network.
As enthusiasm for dumping ice on one another fades with autumn and October brings pervasive pink, I wish that attention would turn to families confronting diseases not as well known as ALS and breast cancer. HOW RARE IS RARE? According … Continue reading
The post No Ice Buckets or Pink Ribbons for Very Rare Genetic Diseases appeared first on PLOS Blogs Network.
Four-year-old Eliza O’Neill’s viral videos, the subject of my last two blog posts, continue to dominate the news media with another appearance on The Today Show June 17. Hopefully, her family’s fight to fund gene therapy for her …
The post Catching Up With 3 Rare Disease Families appeared first on PLOS Blogs Network.