TOPIC: Patients & Members
Today, members of the NORD staff had the opportunity to meet a group of high-school advocates who are already making a difference to help raise awareness for rare diseases. (more…)
Exomes are big news. Sequencing of the protein-encoding part of the genome is increasingly solving medical mysteries in children. (more…)
TOPIC: Advocacy, Industry, Featured News, Patients & Members
Reprinted with permission from FDA Matters: By Steven Grossman
The Orphan Drug Act (ODA) turned 30 this month, demonstrating that good laws really can have an enduring impact. Amidst the celebrations, a reporter asked me a provocative question: Can we afford more orphan drugs costing hundreds of thousands of dollars per year? FDA Matters answered “yes”…. Read More
TOPIC: Patients & Members, Research
Whole genome sequencing (WGS) is the genetic test that determines the order of all 3 billion letters in a person’s DNA, and is a technology that has become well known to the rare disease community. It can reveal not only the genes responsible for production of an abnormal protein associated with a disease, but also the presence of other… Read More
TOPIC: Featured News, Advocacy, Industry
Jack Klugman touched the lives of millions of Americans through his support for what ultimately became the Orphan Drug Act. By portraying on his Quincy, M.E. television show the plight of people with rare diseases with no treatment, Mr. Klugman focused a spotlight on the need for incentives to encourage the development of therapies for small patient populations. He… Read More
