Givlaari, a treatment for acute hepatic porphyria (AHP), was first approved in Europe back in March of 2020. However, approval doesn't mean immediate access unfortunately. It's been nearly two years…
The National Institute for Health and Care Excellence (NICE) has just recommended a gene silencing treatment for acute hepatic porphyria (AHP), a rare disease. AHP AHP is a term for…
Claire Richmond lives with acute hepatic porphyria (AHP). She searched for 19 years to find answers to her unexplained pain. She wasn't diagnosed until she was 32. She recently had…
When a 23-year-old woman, who was 6 weeks pregnant, first appeared at the doctor, she was presenting with non-specific symptoms: pale eyelids, abdominal pain, intermittent cramping, and generalized arm and…
A recent study, titled "Patient and caregiver experiences of living with acute hepatic porphyria in the UK: a mixed method study," investigated the disease burden faced by AHP patients and…
According to BioSpace, a consulting firm called Fact.MR has conducted a study into the market for acute hepatic porphyria (APH). The point of this study was to understand the future…
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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