Drug Repurposing: Saving Rare Disease Patient Lives
WebMD recently carried a story about a young woman who, after nine years of wheelchair confinement caused by a rare metabolic disease with no name, was able to walk…
WebMD recently carried a story about a young woman who, after nine years of wheelchair confinement caused by a rare metabolic disease with no name, was able to walk…
The new nonprofit Every Cure has been created on the basis that every drug currently on the market effects multiple areas in the body. This premise has been proven…
According to a story from Yahoo! Finance, EUSA Pharma Limited and BeiGene, Ltd. have recently announced that their Biologics License Application (BLA) has been accepted by the China National Medical…
10-year-old Joey Koslowski doesn't let the small...err, the big stuff get him down. According to MyCentralJersey, the boy is "the youngest child to have the largest mass in the world" caused…
Not many people can say they’ve dedicated their life to curing the disease that could lead to their death, but David Fajgenbaum certainly can. In a fascinating Science Magazine article, David…
It is hard to study rare diseases because there are so few people with them. Patients are scattered all over the world. Many are children. Many cannot travel. How to make…
Welcome Back Patient Worthians! Acromegaly patients will be interested to see some new research happening in the name of rare disease. Speaking of research, there is a Castleman's event coming up…