According to a story from news-medical.net, a results of a recent study could help accelerate the development of therapies for liver diseases based on RNA interference (RNAi). This study demonstrated…
Earlier this week, a press release from Alnylam Pharmaceuticals, Inc, announced the that the phase 3 study of their experimental acute hepatic porphyria drug Givosiran yielded positive results. According to the…
Thanks to a grant from Worldwide Cancer Research, as well as support from various other organizations, researchers at UCLA recently uncovered a new, faster, easier, and more personalized way to…
Happy Rare Disease Week! This week has been all about rare diseases. Today, we're highlighting an article from a PW contributor with FAP, followed by a story describing some shortcomings…
Cystic Fibrosis Cystic fibrosis (CF) is a rare disease caused by the lack (or malfunctioning) of the cystic fibrosis transmembrane conductance regulator (CTFR) protein. This occurs when children inherit a mutated…
According to a story from BioPortfolio, the biopharmaceutical company Acceleron Pharma Inc., recently announced the release of results from a Phase 2 study which tested the drug luspatercept as a…
According to a story from Parkinson's News Today, a recent study published in the New England Journal of Medicine highlights the key weakness of Levodopa, one of the most common treatments…
According to a story from BioPortfolio, the keto-medical nutrition company Disruptive Nutrition recently announced that the first patient had been enrolled in a research study which will test a unique…
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.
