Category Archives: NORD
Hannah’s 2016: From Curling Toes to Gene Therapy
Posted by clinical trials, featured, GAN, gene therapy, giant axonal neuropathy, Hannah Sames, Lori Sames, NORD, physical therapy, Steven Gray, Xenokeryx
inUnderstudy Gene Offers Hope for Spinal Muscular Atrophy
I began writing about genetics decades ago, and the best thing about getting older is witnessing the development of targeted treatments for single-gene diseases that I never thought would happen. But it is happening, for cystic fibrosis, Duchenne muscular dystrophy, … Continue reading
The post Understudy Gene Offers Hope for Spinal Muscular Atrophy appeared first on PLOS Blogs Network.
Catching Up With 3 Rare Disease Families
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.
Eliza’s Journey: Part 2
Gene therapy is, finally, about to take off! The feeling was palpable at the American Society of Gene and Cell Therapy annual meeting a few weeks ago. It’s been nearly a quarter century since the first experiment in humans.
The drug …
The post Eliza’s Journey: Part 2 appeared first on PLOS Blogs Network.
Posted by ASGCT, gene therapy, Lysogene, NORD, rare disease, Sanfilippo syndrome
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