Related references
Note: Only part of the references are listed.Pharmacokinetics and Pharmacodynamics of the Small Interfering Ribonucleic Acid, Givosiran, in Patients With Acute Hepatic Porphyria
Sagar Agarwal et al.
CLINICAL PHARMACOLOGY & THERAPEUTICS (2020)
A Pharmacological Chaperone Therapy for Acute Intermittent Porphyria
Helene J. Bustad et al.
MOLECULAR THERAPY (2020)
Real-world annualized healthcare utilization and expenditures among insured US patients with acute intermittent porphyria (AIP) treated with hemin
Barbara Blaylock et al.
JOURNAL OF MEDICAL ECONOMICS (2020)
Pathogenesis and clinical features of the acute hepatic porphyrias (AHPs)
Herbert L. Bonkovsky et al.
MOLECULAR GENETICS AND METABOLISM (2019)
Phase 1 Trial of an RNA Interference Therapy for Acute Intermittent Porphyria
Eliane Sardh et al.
NEW ENGLAND JOURNAL OF MEDICINE (2019)
Current and innovative emerging therapies for porphyrias with hepatic involvement
Antonio Fontanellas et al.
JOURNAL OF HEPATOLOGY (2019)
Benefits of prophylactic heme therapy in severe acute intermittent porphyria
Pradeep Yarra et al.
MOLECULAR GENETICS AND METABOLISM REPORTS (2019)
From a dominant to an oligogenic model of inheritance with environmental modifiers in acute intermittent porphyria
Hugo Lenglet et al.
HUMAN MOLECULAR GENETICS (2018)
Systemic messenger RNA as an etiological treatment for acute intermittent porphyria
Lei Jiang et al.
NATURE MEDICINE (2018)
Acute Hepatic Porphyrias: Recommendations for Evaluation and Long-Term Management
Manisha Balwani et al.
HEPATOLOGY (2017)
Porphyria
D. Montgomery Bissell et al.
NEW ENGLAND JOURNAL OF MEDICINE (2017)
Emerging therapies for acute intermittent porphyria
Antonio Fontanellas et al.
EXPERT REVIEWS IN MOLECULAR MEDICINE (2016)
Acute Intermittent Porphyria: Predicted Pathogenicity of HMBS Variants Indicates Extremely Low Penetrance of the Autosomal Dominant Disease
Brenden Chen et al.
HUMAN MUTATION (2016)
Phase I open label liver-directed gene therapy clinical trial for acute intermittent porphyria
Delia D'Avola et al.
JOURNAL OF HEPATOLOGY (2016)
Preclinical Development of a Subcutaneous ALAS1 RNAi Therapeutic for Treatment of Hepatic Porphyrias Using Circulating RNA Quantification
Amy Chan et al.
MOLECULAR THERAPY-NUCLEIC ACIDS (2015)
Acute Porphyrias in the USA: Features of 108 Subjects from Porphyrias Consortium
Herbert L. Bonkovsky et al.
AMERICAN JOURNAL OF MEDICINE (2014)
The incidence of inherited porphyrias in Europe
George Elder et al.
JOURNAL OF INHERITED METABOLIC DISEASE (2013)
Porphyrin and Heme Metabolism and the Porphyrias
Herbert L. Bonkovsky et al.
COMPREHENSIVE PHYSIOLOGY (2013)
Molecular enzymology of 5-Aminolevulinate synthase, the gatekeeper of heme biosynthesis
Gregory A. Hunter et al.
BIOCHIMICA ET BIOPHYSICA ACTA-PROTEINS AND PROTEOMICS (2011)
Recommendations for the diagnosis and treatment of the acute porphyrias
KE Anderson et al.
ANNALS OF INTERNAL MEDICINE (2005)
An alternatively-spliced exon in the 5′-UTR of human ALAS1 mRNA inhibits translation and renders it resistant to haem-mediated decay
AG Roberts et al.
FEBS LETTERS (2005)
Alternative splicing and tissue-specific transcription of human and rodent ubiquitous 5-aminolevulinate synthase (ALAS1) genes
AG Roberts et al.
BIOCHIMICA ET BIOPHYSICA ACTA-GENE STRUCTURE AND EXPRESSION (2001)
Share Paper
