Hope everyone is having a wonderful Wednesday! The weather where we are is freezing this week-- but that's a perfect excuse to sit down with a few of our top…
Danon disease is a rare condition caused by a mutated LAMP-2 gene. This gene has three different forms- LAMP-2A, 2B, and 2C. Depending in which variant is mutated, individuals with…
According to a story from BioPortfolio, the biotechnology company Vertex Pharmaceuticals recently announced that the European Commission has approved a label extension for the company's cystic fibrosis drug Orkambi. Under…
aTTP aTTP, or acquired thrombotic thrombocytopenia purpura, is an immune condition which results in severe Thrombocytopenia. Thrombocytopenia means the body doesn't have enough platelets in the blood. In aTTP, it's caused…
According to a story from news-medical.net, a recent study has a found that a hidden disease called hemochromatosis could be a major risk factor for several different joint diseases and…
Pharnext SA, a biopharmaceutical company, recently announced results in a PLOS ONE article entitled "Early short-term PXT3003 combinational therapy delays disease onset in a transgenic rat model of Charcot-Marie-Tooth disease 1A…
According to a publication from Stat, scientists have high hopes about "virtual repurposing" cutting drug production costs. It could also potentially mean more treatments on the market faster. Virtual Repurposing…
According to a report from ITV, a five-year-old boy from southern England has made the trip across the Bay of Biscay to Barcelona, Spain, where he is receiving an experimental…
Sign Up With a Patient Worthy Account and Share Your Rare Story
- OR -
Make a difference, share your experiences and get paid. Opt-in and join Patient Worthy's panel for paid opportunities such as surveys, market research, patient advisory panels and more.
- OR -
Make a difference, share your experiences and get paid. Opt-in and join Patient Worthy's panel for paid opportunities such as surveys, market research, patient advisory panels and more.
