EXPLORE: A Prospective, Multinational, Natural History Study of Patients with Acute Hepatic Porphyria with Recurrent Attacks.
Laurent GouyaPaolo VenturaManisha BalwaniD Montgomery BissellDavid C ReesUlrich StölzelJohn D PhillipsRaili KauppinenJanneke G LangendonkRobert J DesnickJean-Charles DeybachHerbert L BonkovskyCharles ParkerHetanshi NaikMichael BadmintonPenelope E SteinElisabeth MinderJerzy WindygaRadan BruhaMaria Domenica CappelliniEliane SardhPauline HarperSverre SandbergAasne K AarsandJanice AndersenFélix AlegreAneta IvanovaNeila TalbiAmy ChanWilliam QuerbesJohn KoCraig PenzShangbin LiuTim LinAmy SimonKarl E AndersonPublished in: Hepatology (Baltimore, Md.) (2019)
Patients experienced attacks often requiring treatment in a health care facility and/or with hemin, as well as chronic symptoms that adversely influenced day-to-day functioning. In this patient group, the high disease burden and diminished quality of life highlight the need for novel therapies.