Promising results for new acute porphyria treatment
[PRESS RELEASE 2019-02-06] Acute porphyria is a group of uncommon diseases that can cause severe, potentially life-threatening attacks of abdominal pain, nausea, vomiting and paralysis. Liver transplantation is currently the only effective treatment available for the most seriously afflicted patients. A clinical trial conducted in collaboration with researchers at Karolinska Institutet in Sweden now shows that a new drug candidate can prevent attacks in these patients. The study is published in The New England Journal of Medicine.Porphyria is caused by different inherited disorders in the