Santa brought these fun beads to Isaac for Christmas and I just remembered them and hydrated a few.
He was interested in the texture and temperature. 26 more words
Every time I visit Steve Gray and the University of North Carolina Gene Therapy Center, I want to run home and immediately write a blog post and email all of my media contacts and friends in the rare disease community and tell them what’s happening in a lab two hours from my hometown. 445 more words
Rare disease patient families are setting up not-for-profit organizations, leading the way for funding research and accelerating the drug approval process. This Blog Post looks at two rare disease advocacy groups that have received FDA Orphan Drug Designation (ODD) for novel gene therapies: 527 more words