• Givosiran reduces neurotoxic intermediates in acute hepatic porphyria

    3 days ago - By Healio

    PARIS - Givosiran, an investigational RNA interference therapeutic, was generally well-tolerated and reduced aminolevulinic acid and porphobilinogen accumulation in patients with acute hepatic porphyria and ameliorate disease manifestations, according to a presentation at the International Liver Congress 2018.
    “Acute intermittent porphyria is a rare disorder which is caused by mutations in heme synthesis,” Eliane Sardh, MD, PhD, from the Karolinska University Hospital in Sweden, told Healio Gastroenterology and Liver Disease. “I have presented new data on a new
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