Patricia Inácio, PhD,  science writer—

Acute intermittent porphyria (AIP) patients who have recurrent attacks are more likely to be sicker and unemployed, yet face higher medical costs than patients with occasional attacks, researchers in The Netherlands report. Their study, “Medical and financial burden of acute intermittent porphyria,” was published in the Journal of…

The U.S. Food and Drug Administration (FDA) has granted orphan drug designation to AMP-L2.7.D7 (ciclopirox) for treatment of Congenital Erythropoietic Porphyria (also known as Gunther’s disease). AMP-L2.7.D7, developed by Atlas Molecular Pharma, is a pharmacological chaperone — a molecule that binds to uroporphyrinogen III synthase, the…