Cargando…

Disease burden in patients with acute hepatic porphyria: experience from the phase 3 ENVISION study

BACKGROUND: Acute hepatic porphyria (AHP) is a family of four rare genetic diseases, each involving deficiency in a hepatic heme biosynthetic enzyme. Resultant overproduction of the neurotoxic intermediates δ-aminolevulinic acid (ALA) and porphobilinogen (PBG) leads to disabling acute neurovisceral...

Descripción completa

Detalles Bibliográficos
Autores principales:	Wang, Bruce, Ventura, Paolo, Takase, Kei-ichiro, Thapar, Manish, Cassiman, David, Kubisch, Ilja, Liu, Shangbin, Sweetser, Marianne T., Balwani, Manisha
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	BioMed Central 2022
Materias:	Research
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9419398/ https://www.ncbi.nlm.nih.gov/pubmed/36028858 http://dx.doi.org/10.1186/s13023-022-02463-x

Ejemplares similares

Efficacy and safety of givosiran for acute hepatic porphyria: 24‐month interim analysis of the randomized phase 3 ENVISION study
por: Ventura, Paolo, et al.
Publicado: (2021)

EXPLORE B: A prospective, long‐term natural history study of patients with acute hepatic porphyria with chronic symptoms
por: Cassiman, David, et al.
Publicado: (2022)

Porphyrias in the Age of Targeted Therapies
por: Erwin, Angelika L., et al.
Publicado: (2021)

AGA Clinical Practice Update on Diagnosis and Management of Acute Hepatic Porphyrias: Expert Review
por: Wang, Bruce, et al.
Publicado: (2023)

Acute Hepatic Porphyria
por: Bissell, D. Montgomery, et al.
Publicado: (2015)

Mechanisms of Neuronal Damage in Acute Hepatic Porphyrias
por: Ricci, Andrea, et al.
Publicado: (2021)

Endothelial Dysfunction in Acute Hepatic Porphyrias
por: Ricci, Andrea, et al.
Publicado: (2022)

EXPLORE: A Prospective, Multinational, Natural History Study of Patients with Acute Hepatic Porphyria with Recurrent Attacks
por: Gouya, Laurent, et al.
Publicado: (2019)

Kidney Involvement in Acute Hepatic Porphyrias: Pathophysiology and Diagnostic Implications
por: Ricci, Andrea, et al.
Publicado: (2021)

Iron in Porphyrias: Friend or Foe?
por: Buzzetti, Elena, et al.
Publicado: (2022)

Acute Hepatic Porphyrias: Review and Recent Progress
por: Wang, Bruce, et al.
Publicado: (2018)

Detect Acute Porphyrias in Emergency Departments (DePorED) – a pilot study
por: Diehl-Wiesenecker, Eva, et al.
Publicado: (2023)

Envisioning information
por: Tufte, Edward R., 1942-
Publicado: (1990)

Envisioning information
por: Tufte, Edward Rolf
Publicado: (2003)

ENVISION Project
por: Ballantine, A, et al.
Publicado: (2011)

Medical and financial burden of acute intermittent porphyria
por: Neeleman, Rochus A., et al.
Publicado: (2018)

Estimating the broader fiscal consequences of acute hepatic porphyria (AHP) with recurrent attacks in Belgium using a public economic analytic framework
por: Connolly, Mark P., et al.
Publicado: (2021)

Greater disease burden of variegate porphyria than hereditary coproporphyria: An Israeli nationwide study of neurocutaneous porphyrias
por: Kaftory, Ran, et al.
Publicado: (2021)

Acute hepatic porphyria and hepatocellular carcinoma.
por: Kauppinen, R., et al.
Publicado: (1988)

Evaluating the Patient Reported Outcomes Measurement Information System Scales in Acute Intermittent Porphyria
por: Naik, Hetanshi, et al.
Publicado: (2019)

Envisioning architecture : an analysis of drawing /
por: Fraser, Iain
Publicado: (1994)

Envisioning the future of reference
por: Zabel, Diane, et al.
Publicado: (2020)

Envisioning the expertise of the future
por: Naydenova, Svetla, et al.
Publicado: (2019)

Editorial: Envisioning the Future of Industrial Bioprocesses Through Biorefinery
por: Pereira, Jorge F. B., et al.
Publicado: (2021)

Challenges in diagnosis and management of acute hepatic porphyrias: from an uncommon pediatric onset to innovative treatments and perspectives
por: Marcacci, Matteo, et al.
Publicado: (2022)

Pathogenesis of acute encephalopathy in acute hepatic porphyria
por: Pischik, Elena, et al.
Publicado: (2023)

THE PORPHYRIAS
Publicado: (1981)

Envisioning emerging frontiers on human gut microbiota and its applications
por: Ventura, Marco, et al.
Publicado: (2020)

ENLIGHT envisions its future
por: Antonella Del Rosso
Publicado: (2015)

C2 Re-envisioned
por: Vassiliou, Marius, et al.
Publicado: (2014)

Envisioning the futures of cultural tourism
por: Matteucci, Xavier, et al.
Publicado: (2022)

Envisioning social drones in education
por: Johal, Wafa, et al.
Publicado: (2022)

Envisioning others : race, color, and the visual in Iberia and Latin America /
Publicado: (2016)

PB2562: STUDY DESIGN OF THE AURORA TRIAL: A PHASE 2, RANDOMIZED, DOUBLE-BLIND, PLACEBO-CONTROLLED TRIAL OF BITOPERTIN IN ERYTHROPOIETIC PROTOPORPHYRIA
por: Levy, Cynthia, et al.
Publicado: (2023)

Hepatitis C- and HIV-induced porphyria cutanea tarda
por: Quansah, Raphael, et al.
Publicado: (2014)

The association between chemical-induced porphyria and hepatic cancer
por: Smith, Andrew G., et al.
Publicado: (2018)

Variegate Porphyria Triggered by Acute Hepatitis A Infection
por: Micallef, Daniel, et al.
Publicado: (2020)

Acute Hepatic Porphyria: Pathophysiological Basis of Neuromuscular Manifestations
por: de Souza, Paulo Victor Sgobbi, et al.
Publicado: (2021)

Pain in acute hepatic porphyrias: Updates on pathophysiology and management
por: Kazamel, Mohamed, et al.
Publicado: (2022)

Clinical features of Japanese patients with acute hepatic porphyria
por: Horie, Yutaka, et al.
Publicado: (2022)

Cannot write session to /tmp/vufind_sessions/sess_obvskmvdf6nbjdtlo43jii18th