While only 3 small interfering RNA (siRNA)-based therapies have been approved, the market is poised to expand with 7 other candidates in phase 3 trials.
Based on the findings, the researchers signal that family screening following a sentinel case may reap benefits for patients with acute hepatic porphyria (AHP).
A group of researchers discuss the clinical features and diagnosis of acute hepatic porphyrias (AHP) as well as their take on the approval of the first small interfering RNA (siRNA)-based therapy for the treatment of these patients.
Study results published in PLoS One strongly suggest machine learning can be an effective approach to identify patients who should receive a diagnostic biochemical test to screen for acute hepatic porphyria.
Patients with symptomatic acute hepatic porphyria were found to be at greater risk of accessing long-term sick leave and disability pension compared with the general population, but were not shown to be at an increased overall risk of premature death.