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Porphyrias
Clinical trial pipeline · Data from ClinicalTrials.gov
See which Porphyrias trials may be worth asking aboutClinical trial pipeline · Data from ClinicalTrials.gov
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The objective of this protocol is to conduct a longitudinal multidisciplinary investigation of the human porphyrias including the natural history, morbidity, pr…
The purpose of this study is to collect information about the effects of EPP/XLP in adults and adolescents. This is an observational study in which participants…
This global patient registry is being conducted to characterize the natural history and real-world clinical management of patients with AHP, and to further char…
The main aim of this clinical trial is to learn about the effect of carbohydrate-rich diet as a treatment for AIP (acute intermittent porphyria). Aim: Investig…
This is a study to investigate the effect of oral ATL-001 (ciclopirox) in CEP (Congenital Erythropoietic Porphyria) patients. During the study, it will be measu…
This study proposes to identify the predisposing/protective modifying genes that underlie the acute attacks in symptomatic patients with Acute Intermittent Porp…
Erythropoietic protoporphyria (EPP) and X-linked protoporphyria (XLP) result from genetic defects of heme biosynthesis that cause life-long, painful cutaneous s…
This is an open-label, long-term extension study to investigate the safety, tolerability and efficacy of DISC-1459 in participants with EPP.