About 225 results

ALLMedicine™ Porphyria Center

Research & Reviews  88 results

Hyperhomocysteinemia in acute hepatic porphyria (AHP) and implications for treatment wi...
Expert Review of Gastroenterology & Hepatology; Ventura P, Sardh E et. al.

Aug 6th, 2022 - : Homocysteine is a sulfur-containing amino acid formed in the intermediary metabolism of methionine. Amino acid metabolism and heme biosynthesis pathways are complexly intertwined. Plasma homocysteine elevation, hyperhomocysteinemia (HHcy), has b...

Patient Perspective on Acute Hepatic Porphyria with Sporadic Attacks: A Chronic Disease...
Advances in Therapy; Wheeden K, Lyon Howe D et. al.

Jul 31st, 2022 - Acute hepatic porphyria (AHP) is a family of rare metabolic diseases characterized by potentially life-threatening acute attacks and, in some patients, chronic debilitating symptoms. While patients with frequent or recurrent attacks (three or more...

Biallelic hydroxymethylbilane synthase inactivation defines a homogenous clinico-molecu...
Journal of Hepatology; Molina L, Zhu J et. al.

Jun 1st, 2022 - Acute intermittent porphyria (AIP), caused by heterozygous germline mutations of the heme synthesis pathway enzyme HMBS (hydroxymethylbilane synthase), confers high risk of hepatocellular carcinoma (HCC) development. Yet, the role of HMBS in liver...

Acute intermittent porphyria: is oseltamivir safe in these patients?
Clinical Medicine (London, England); Amador AP, Cordero AS et. al.

May 19th, 2022 - A 40-year-old man attended the emergency room with abdominal pain and inappropriate behaviour associated with stress, and the consumption of alcohol and cannabis. Examination revealed hypertension (155/100 mmHg), tachycardia (95 beats per minute),...

Update on the diagnosis and management of the autosomal dominant acute hepatic porphyrias.
Journal of Clinical Pathology; Schulenburg-Brand D, Stewart F et. al.

May 19th, 2022 - The autosomal dominant acute hepatic porphyrias (AHPs), acute intermittent porphyria, hereditary coproporphyria (HCP) and variegate porphyria (VP), are low penetrance adult onset disorders caused by partial deficiency of enzymes of haem biosynthes...

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Drugs  1 results

Givlaari - givosiran sodium injection, solution-Alnylam Pharmaceuticals, Inc.

Oct 14th, 2021 - GIVLAARI is indicated for the treatment of adults with acute hepatic porphyria (AHP). GIVLAARI is an aminolevulinate synthase 1-directed small interfering RNA indicated for the treatment of adults with acute hepatic porphyria (AHP). (1)

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News  1 results

EMA Panel Backs Givlaari, First Treatment for Acute Hepatic Porphyria

Jan 31st, 2020 - The European Medicines Agency's (EMA) Committee for Medicinal Products for Human Use (CHMP) recommended marketing authorization for givosiran (Givlaari, Alnylam), the first treatment for acute hepatic porphyria (AHP) for patients aged 12 years and...

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