News

A young woman with acute intermittent porphyria (AIP) experienced neuropathic pain — a type of pain caused by nerve damage — after receiving a liver transplant, according to a report from investigators in Brazil. For the first five months following the procedure, the woman experienced no symptoms, but then…

Women with acute intermittent porphyria (AIP) receiving once-weekly preventive treatment with heme arginate — given as an intravenous or into-the-vein infusion — had fewer and less severe attacks, a small study found. Indeed, these AIP patients saw a reduction in acute attacks by more than 80% with the use…

Alpha-lipoic acid, an insulin sensitizer, may help improve the breakdown and use of glucose, or blood sugar, in the liver of patients with acute intermittent porphyria (AIP), according to a recent cell-based study. The findings showed that alpha-lipoic acid is “attractive for the management of metabolic disorders, as it…

Acute intermittent porphyria (AIP) attacks can be stopped by regular peritoneal dialysis, a treatment that eliminates waste products by rinsing the inner abdomen, as reported in a case study. According to researchers, this study suggests that peritoneal dialysis may be used both as a preventive treatment and during AIP…

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 The announcement comes in the wake of a draft guidance…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…