Vertex Pharmaceuticals announced the submission of a New Drug Application (NDA) to the FDA for the VX-445 (elexacaftor), tezacaftor and ivacaftor triple combination regimen for treating Cystic Fibrosis (CF). This…
The Mental Health Rare Genetic Disease Network (MHRGDN) is a newly launched system, developed by the National Institute of Mental Health. The MHRGDN spans 15 research sites across the country…
Learning that you or a loved one has a rare genetic disease can be scary. Doctors often are not very well versed in these types of disorders and may require…
Cyxone has just announced that the first person has been dosed in their Phase 1 clinical trial for Multiple Sclerosis (MS). This trial is investigating the effect of T20K. It…
According to a story from BioPortfolio, the company Novocure has recently announced that Medicare has released final local coverage determinations for its product Optune. Optune is used to treat patients…
No Cure, No Pay Emile Voest, an oncologist in the Netherlands, conceived the idea of a no cure, no pay model. After witnessing the exorbitant cost of drugs, some of…
Throughout my daughter Kelley’s 45-year life with a rare disease, there were numerous times that we needed to either call 911 or take her to the Emergency Department at a…
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