People with acute hepatic porphyria (AHP) tend to have higher-than-normal levels of markers for kidney injury, new research suggests. The findings were shared in a virtual poster, titled “Renal Injury Biomarkers Are Elevated in Acute Hepatic Porphyria,” presented at the American Society of Nephrology Kidney Week…
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 The announcement comes in the wake of a draft guidance…
The Canadian Agency for Drugs and Technology in Health, called CADTH, has recommended that Givlaari (givosiran) be reimbursed by public plans — if certain conditions are met — when it’s used to treat adults with acute hepatic porphyria (AHP). One condition, according to CADTH, is that the therapy’s…
Global Genes has partnered with the Rare Disease Diversity Coalition (RDDC) to advance health equity for rare disease patients and caregivers in underrepresented communities of color. “For rare disease patients, there are many challenges — and for people of color with a rare disease, these challenges are compounded…
Long-term treatment with Givlaari (givosiran) is safe and provides sustained clinical benefits to people with acute hepatic porphyria (AHP), according to an interim, two-year analysis of a Phase 3 trial’s ongoing extension, presented at a science conference. These data are in line with previous trial findings, showing that the…
The Rare Disease Diversity Coalition (RDDC) awarded $600,000 in grants to ease the disparities faced by rare disease patients of color. These Impact Rare Disease Solution grants will go five RDDC steering committee working groups, which aim to identify problems for rare disease communities and advocate for solutions. The five…
Recurrent attacks in people with acute hepatic porphyria (AHP) are associated with lower lifetime earnings and pose a higher burden to public health, particularly in disability and healthcare costs, a Belgian study reports. Indeed, the costs of lifetime healthcare for patients in Belgium who are unable to work due…
A young pregnant woman was diagnosed with acute hepatic porphyria (AHP) after her urine was found to glow pink under ultraviolet (UV) light, as described in a recent case report. The woman’s symptoms were general, which often makes an early diagnosis difficult. A “simple method like urine examination under ultraviolet…
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…
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