Erythropoietic protoporphyria is a genetic disease in which ferrochelatase is defective. Protoporphyrin accumulates in erythrocytes, leaks into the plasma and results in severe skin photosensitivity.

Study: The histamine pathway is a target to treat hepatic experimental erythropoietic protoporphyria. Image Credit: luchschenF/Shutterstock.com In a recent study published in the Cellular and ...

Disc Medicine has charted a route to market for bitopertin, its drug candidate for rare disease erythropoietic protoporphyria (EPP), after meeting with the FDA. Shares in the company gained almost ...

Bitopertin is an investigational agent and is not approved for use as a therapy in any jurisdiction worldwide. Disc obtained global rights to bitopertin under a license agreement from Roche in May ...

Erythropoietic protoporphyria (EPP), including X-linked Protoporphyria (XLP), is a rare, debilitating and potentially life-threatening disease caused by mutations that affect heme biosynthesis ...

Porphyrias are caused by enzymatic dysfunctions in the haem biosynthesis metabolic pathway. 1 Individuals with EPP (erythropoietic protoporphyria) and CEP (congenital erythropoietic porphyria) treated ...

Disc obtained global rights to bitopertin under a license agreement from Roche in May 2021. Erythropoietic protoporphyria (EPP), including X-linked Protoporphyria (XLP), is a rare, debilitating and ...

focusing on Erythropoietic Protoporphyria (EPP) and X-linked Protoporphyria (XLP). This progression surfaced as part of a current report on Form 8-K, offering important insights into the company ...

Inclusion of patients aged 12+ with EPP including X-linked protoporphyria (XLP); and Double-blind, placebo-controlled study with ~150 patients randomized 1:1.