Dersimelagon in Erythropoietic Protoporphyrias

In a phase 2 trial, once-daily oral treatment with dersimelagon safely improved tolerance to sun exposure in patients with erythropoietic protoporphyria and X-linked protoporphyria.

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Bibliographische Detailangaben
Veröffentlicht in:	The New England journal of medicine 2023-04, Vol.388 (15), p.1376-1385
Hauptverfasser:	Balwani, Manisha, Bonkovsky, Herbert L., Levy, Cynthia, Anderson, Karl E., Bissell, D. Montgomery, Parker, Charles, Takahashi, Fumihiro, Desnick, Robert J., Belongie, Kirstine
Format:	Artikel
Sprache:	eng
Schlagworte:	Administration, Oral Data analysis Dermatologic Agents - therapeutic use Dermatology Dermatology General Diaries Endothelium Erythropoietic protoporphyria Genetics Genetics General Heme Humans Hyperpigmentation Infant, Newborn Light - adverse effects Liver Melanocortin Melanocyte-stimulating hormone Neurology Neurosurgery Oral administration Pain Patients Photosensitivity Disorders - etiology Phototoxicity Placebos Prodromal Symptoms Protoporphyria, Erythropoietic - complications Protoporphyria, Erythropoietic - drug therapy Protoporphyrin Quality of Life Receptor, Melanocortin, Type 1 - agonists Skin - drug effects Sun Sunlight Transplants & implants
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Beschreibung
Zusammenfassung:	In a phase 2 trial, once-daily oral treatment with dersimelagon safely improved tolerance to sun exposure in patients with erythropoietic protoporphyria and X-linked protoporphyria.
ISSN:	0028-4793 1533-4406
DOI:	10.1056/NEJMoa2208754