On November 19, 2020, Research!America hosted an informational webinar titled "From Nobel Winning Science to Next Generation Treatment: Tracing the Path of a Rare Disease Breakthrough using RNAi." This webinar focused…
In 2019, Alnylam Pharmaceuticals received FDA approval for Givlaari, the first RNAi therapy for patients with acute hepatic porphyria. Although some were initially worried about the therapy's safety and…
According to a press release from Alnylam Pharmaceuticals, the Massachusetts-based biopharmaceutical company has completed the submission of its New Drug Application (NDA) for givosiran — its experimental acute hepatic porphyria…
According to a story from Healio, there are a number of recent reports of progress in the development of treatments for rare forms of liver disease. Many rare liver diseases…
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…
For people who don't live with chronic painful conditions, such as hepatic porphyria, it's hard to imagine the type of intense pain Colin McEwen was in -- both physically and…
On January 12-14, 2018 leading minds from around the world will convene in Orlando, Florida for the annual Heme Biosynthesis and the Porphyrias Recent Advances Conference. Yes, it's a mouthful,…
Suffering alone with a rare, chronic condition is a terrible thing. But thanks to the National Porphyria Registry people living with this vexing disease have a way to stand up,…
The American Porphyria Foundation has a section on its website dedicated to telling patient stories. It gives voice to people's experiences and allows them to connect with others and share…
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