Long-term treatment with Givlaari (givosiran) lessens rates of porphyria attacks, eases pain, and improves quality of life in individuals with acute hepatic porphyria (AHP), according to two-year interim data from the ENVISION trial. Results were reported in the study, “Efficacy and safety of givosiran for…
By February, eligible acute porphyria patients in England are expected to have access to Givlaari (givosiran) through the country’s National Health Service (NHS), for treating severe, recurrent disease attacks, according to an update from the British Porphyria Association. The announcement comes in the wake of a draft guidance…
Experts will discuss the latest research related to porphyria, as well as current and emerging treatments and management strategies, at an upcoming symposium. The event will include a free patient day, open to both in-person and virtual attendees. “The Porphyrias Symposium … will bring together Porphyrias experts, treating…
Most people with porphyria who responded to a U.S.-based online survey report being generally satisfied with their treatment plan, and many are open to participating in clinical trials. About two-thirds of the 70 surveyed patients said they are content with their quality of life, but just as many are…
The Assistance Fund (TAF) is accepting new eligible patients in the U.S. into its financial aid program for porphyria. The program, which was launched last year, helps individuals to pay for out-of-pocket costs associated with treatment. Financial assistance applies to copays, deductibles, and coinsurance for approved medications; costs…
A baby girl was found to have congenital erythropoietic porphyria, highlighting the importance of closely examining the bone marrow for diagnosing this rare form of porphyria. Her case was described in the study, “Bone marrow erythroid cell inclusions reveal congenital erythropoietic porphyria,” published in the…
Eurordis opened a campaign, called Rare 2030 Action, that is seeking to establish a European action plan for rare diseases to ensure that none of the 30 million people in Europe living with rare diseases are left behind by the start of a new decade. As part of…
Abdominal pain and nausea in a woman having an acute porphyria attack was initially misdiagnosed as a natural response to the early stages of a pregnancy, a recent case study reported. Her case, its authors noted, highlighted the importance of performing urine screening tests whenever porphyria is suspected,…
Applications are now open for Find Your Shadow 2021, an adventure program from Shadow Jumpers that aims to help children with erythropoietic protoporphyria (EPP) try new things they otherwise would not do because of their disease. EPP is a type of cutaneous porphyria — a porphyria that…
Note: This story was updated on Feb. 2, 2021, to note that cutaneous symptoms are more common in VP and HCP than in AIP or ADP. The American Porphyria Foundation is joining with others in asking people with acute hepatic porphyria to participate in the Porphyria Worldwide Patient Experience Research (POWER)…
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