Compassion [kuhm-pash-uhn] noun A feeling of deep sympathy and sorrow for another who is stricken by misfortune, accompanied by a strong desire to alleviate the suffering. Compassion Corner is a…
In a recent article at Porphyria News, Claire Richmond shares how writing has helped her process her diagnosis of porphyria and to feel connected to the porphyria community. Claire Richmond’s…
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…
In a recent article from Porphyria News, Claire Richmond describes how she lives with acute hepatic porphyria (AHP), a rare disease that requires long-term management. Acute Hepatic Porphyria (AHP)…
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…
According to an article published by Alnylam Pharmaceuticals, a recent survey has provided insight into the burden experienced by acute hepatic porphyria (AHP) patients. Titled the POWER (POrphyria Worldwide Patient…
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GIVLAARI has already received approval for the treatment of acute hepatic porphyria (AHP) in Canada, according to an article from Nasdaq. Now, the Canadian Agency for Drugs and Technologies in…
Welcome to Study of the Week from Patient Worthy. In this segment, we select a study we posted about from the previous week that we think is of particular interest…
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…
Claire Richmond can be considered a very experienced patient. It took Claire nineteen years to receive a diagnosis for her rare disease. During that time, she had been dismissed…
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…
My name is Megan and I live with acute hepatic porphyria (AHP). My health journey began with the arrival of puberty. I started to experience terrible nausea and abdominal pain…
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…
As originally reported in Biospace, there’s good news today for adult patients with acute hepatic porphyria in Europe, a genetic disorder which causes pain and paralysis. The European Commission has…
As originally reported in CheckOrphan, there's good news today for adult patients with acute hepatic porphyria, a genetic disorder which causes pain and paralysis. The Food and Drug Administration (FDA)…
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…
According to a story from wtvbam.com, the drug development company Alnylam Pharmaceuticals has recently stated that it is looking less likely that its gene silencing therapy givosiran will be getting…
Topline results have been shared from a Phase 3 study of the investigational drug givosiran in patients with acute hepatic porphyria. For more detailed information you can view the source…
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