Treatment with bitopertin, an oral therapy from Disc Medicine, led to increased sunlight tolerance and improved quality of life for people with erythropoietic protoporphyria (EPP) in the Phase 2 BEACON trial, preliminary data show. Levels of protoporphyrin IX (PPIX), the molecule that builds up to toxic levels in EPP…
Women with acute hepatic porphyria (AHP) were at a higher risk of developing high blood pressure and diabetes during pregnancy than their healthy counterparts, and their babies were more likely to be born small, according to a Swedish study. Those with acute intermittent porphyria (AIP) — the most common…
MT-7117 (dersimelagon), an investigational oral therapy from Mitsubishi Tanabe Pharma, safely reduced sunlight sensitivity and improved quality of life in people with erythropoietic protoporphyria (EPP) and X-linked protoporphyria (XLP) — two types of cutaneous porphyria, which primarily affects the skin — in a Phase 2 trial. Findings from…
Enrollment is still underway in the Phase 2 clinical trial AURORA for bitopertin as a potential disease-modifying treatment for erythropoietic protoporphyria (EPP). AURORA (NCT05308472), which launched in October, is seeking to enroll up to 75 adults with EPP at several sites in the U.S. Meanwhile, data from…
A young woman in France experienced a stroke as a reversible neurological consequence of an acute intermittent porphyria (AIP) attack that blocked blood flow to the brain, according to a recent case report. “The findings in our patient document reversible arterial narrowing” that caused the blood flow to be…
Women with acute hepatic porphyria (AHP) who experience sporadic attacks reported facing chronic symptoms and significant disruptions to their daily activities around each attack, according to a recent study. During patient interviews, these women also frequently described symptom “flares” that they did not consider to be an acute porphyria…
Disc Medicine has launched a Phase 2 trial of bitopertin, an investigational oral treatment for erythropoietic porphyrias. Dubbed BEACON, the open-label trial will investigate the safety, tolerability, and efficacy of multiple doses of bitopertin in about 20 patients with erythropoietic protoporphyria (EPP) and X-linked protoporphyria (XLP) at sites…
People with porphyria have a 5% increased risk of developing primary liver cancer compared with the general population, according to a recent review study. The findings highlight the importance of regular cancer screenings in porphyria patients, especially those older than 50 years, researchers noted. The study, “…
Undiagnosed hereditary coproporphyria (HCP) was the cause of psychiatric symptoms, including aggressive behaviors, delusions, and visual hallucinations, in a 66-year-old-man initially misdiagnosed with schizoaffective disorder, according to a recent case report. “Nonspecific symptoms of acute porphyria can mimic many other diseases and lead to delayed diagnosis or misdiagnosis,” the…
Phlebotomy, or regular rounds of blood letting, eased symptoms of liver injury in three people with erythropoietic protoporphyria (EPP), according to a small study. Besides supporting the utility of phlebotomy in treating this patient population, study findings suggest a potential role for the ABCG2 protein in the mechanisms that…
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