Article

Acitretin mitigates uroporphyrin-induced bone defects in congenital erythropoietic porphyria models

2021-03-30

Abstract excerpt

<h4>ABSTRACT</h4> Congenital erythropoietic porphyria (CEP) is a rare genetic disorder leading to accumulation of uro/coproporphyrin-I in tissues due to inhibition of uroporphyrinogen-III synthase. Clinical manifestations of CEP include bone fragility, severe photosensitivity and photomutilation. Currently there is no specific treatment for CEP, except bone marrow transplantation, and there is an unmet need for t...

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Literature Corpus work: 4e63394d-cf08-5fd6-a9e4-32684d2ee1f4
DOI: 10.1101/2021.03.30.437658

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