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Results Demonstrate Potential for Givosiran as a Treatment for Acute Hepatic Porphyria

Rare Disease Report - Oct 1, 2018
Givosiran, an investigational RNA interference (RNAi) therapy, significantly reduced urinary aminolevulinic acid (ALA), a disease biomarker reasonably likely to predict clinical benefit for the treatment of acute hepatic porphyria (AHP), according to a ...

Existing Drug Could Treat Porphyria

Technology Networks - Sep 19, 2018
An existing antifungal drug, ciclopirox (CPX), has been shown to improve symptoms associated with congenital erythropoietic porphyria – the rarest type of porphyria. The study was conducted using a mouse model of the disease, the team's findings were ...

Antifungal agent found to be possible treatment for porphyria

Medical Xpress - Sep 24, 2018
A large team of researchers from Spain, France and the U.S. has found that a common antifungal agent might be useful as a treatment for a rare type of porphyria. In their paper published in the journal Science Translational Medicine, the group ...

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Porphyria pathology

Porphyria is a metabolic disease in the synthesis of Heme. Heme is made up of a molecule of protoporphyrin plus an iron ion Fe(2 ). When the enzymes that ...

Porphyrias | Dr. Tony Talebi lectures on Porphyrias

Dr. Tony Talebi lectures on porphyrias. This lectures corresponds to the best selling book \

Acute Intermittent Porphyria - Usmle step 1 Biochemistry webinar lecture

The most common of the acute porphyrias, acute intermittent porphyria (AIP) is an autosomal dominant genetic disease marked by deficient levels of the enzyme ...

Acute Intermittent Porphyria

Acute intermittent porphyria is an inherited metabolic disorder which affects 1 to 5 per million of the American population predominantly women between 18-40 ...