Marisa Wexler, MS, senior science writer —

The European Commission is expected to propose a new governing framework for health data next month, called the European Health Data Space (EHDS), with the aim of connecting national health systems to facilitate secure and efficient transfer of data across systems in different European nations. The move is expected to…

The case of a man with porphyria cutanea tarda (PCT) that damaged his eyes — a rare manifestation of the disease that progressed to the point that it threatened his eyesight — was described in a report from India. The report, “Sight-threatening progressive corneo-scleral involvement in porphyria…

Abnormal fatty lesions in the liver, detected via ultrasound, may be critical for diagnosing some cases of porphyria cutanea tarda (PCT), the most common form of porphyria, according to a study. Its authors suggest that clinicians should use urinary tests to check for PCT in patients with unexplained…

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 The announcement comes in the wake of a draft guidance…

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…