Madigan, K.E.; Rudnick, S.R.; Agnew, M.A.; Urooj, N.; Bonkovsky, H.L. Illuminating Dersimelagon: A Novel Agent in the Treatment of Erythropoietic Protoporphyria and X-Linked Protoporphyria. Pharmaceuticals2024, 17, 31.
Madigan, K.E.; Rudnick, S.R.; Agnew, M.A.; Urooj, N.; Bonkovsky, H.L. Illuminating Dersimelagon: A Novel Agent in the Treatment of Erythropoietic Protoporphyria and X-Linked Protoporphyria. Pharmaceuticals 2024, 17, 31.
Madigan, K.E.; Rudnick, S.R.; Agnew, M.A.; Urooj, N.; Bonkovsky, H.L. Illuminating Dersimelagon: A Novel Agent in the Treatment of Erythropoietic Protoporphyria and X-Linked Protoporphyria. Pharmaceuticals2024, 17, 31.
Madigan, K.E.; Rudnick, S.R.; Agnew, M.A.; Urooj, N.; Bonkovsky, H.L. Illuminating Dersimelagon: A Novel Agent in the Treatment of Erythropoietic Protoporphyria and X-Linked Protoporphyria. Pharmaceuticals 2024, 17, 31.
Abstract
Background: Erythropoietic protoporphyria (EPP) and X-linked protoporphyria (XLP) are the two most common subtypes within rare genetic photodermatoses, which can lead to incapacitating pain episodes. Methods: We reviewed previous and current treatment options for EPP/XLP, notably with a focus on metabolism, pharmacokinetics, mechanism of action, key clinical trial results, and limitations of Dersimelagon [MT-7117]. Results: In review of treatment modalities for EPP/XLP, Dersimelagon has demonstrated an acceptable safety profile with the ease of once-daily oral dosage. Conclusions: Dersimelagon should be a considered as a potential treatment for EPP/XLP. Ongoing studies will provide additional insights into expanding the treatment options for those suffering from EPP/XLP.
Medicine and Pharmacology, Gastroenterology and Hepatology
Copyright:
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