"Orphan diseases" are medical conditions that affect only a few people in a population. They are called orphan diseases because they often lack attention and investment from pharmaceutical companies and medical researchers. Full definition
Dr. De Vivo emphasized that in developing drugs for orphan diseases such as SMA, collaborative programs are a must, and that «strong relationships with disease advocacy foundations are absolutely key.» (smafoundation.org)
The Jackson Laboratory's Rare and Orphan Disease Center in Bar Harbor is creating mouse models with Talia's very rare genetic disorder in the hopes that scientists can use the mice to find a treatment for the disorder. (mdibl.org)
Experience in orphan disease drug development and an understanding of genetic concepts and molecular biology laboratory techniques is beneficial. (editasmedicine.com)