We hope that everybody is having a great Easter, Passover, or first taste of spring! This week we have a story about sisters with Batten disease who are finally being…
La Jolla Pharmaceutical recently announced a new clinical trial targeting thalassemia. The trial, described as pivotal, investigates a drug referred to as LJPC-401. The treatment is intended to help patients…
As we close out the month of March - it's important to give a little thought to some of the lesser-known rare diseases that are often overlooked. Bleeding disorders is…
According to a story from cancercompass.com, a recent study has demonstrated that income level is a significant factor in determining survival for patients with anal cancer. In general, the study…
According to a story from medicalxpress.com, a recent study suggests that cognitive deficits that can occur as side effects of targeted brain cancer drugs could be successfully reversed with therapy.…
A recent story from The Independent is touting the results of a clinical trial that tested stem cells transplants for multiple sclerosis. The transplant appears to help "reset" the patient's…
According to a story from GlobeNewswire, the biotechnology company argenx announced recently that its investigational therapy ARGX-113 was granted orphan drug status by the European Commission. This designation is based…
The disease may be daunting, but it won't stop young, determined woman Ellis Kerton. According to LincolnshireLive, Ellis learned when she was 19-years-old that she was positive for Huntington's disease.…
According to an article from the Augusta Free Press, a research team from Vanderbilt University and the Virginia Tech Carilion Research Institute has successfully managed to uncover the manner in…
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