Using RNAi to GIVe relief from acute hepatic porphyria

Acute hepatic porphyria (AHP) is a rare genetic disorder caused by mutations that affects haem production, resulting in the accumulation of harmful porphyrin precursors in the liver. These build-ups can cause severe abdominal pain and other debilitating symptoms. Traditional treatments, such as intravenous haemin infusions, can be slow and may lead to unwanted side effects. In this video, we explore Givosiran, a revolutionary RNA interference therapy designed to target and silence the ALAS1 gene in the liver. By doing so, Givosiran effectively reduces the levels of toxic metabolites and helps prevent painful attacks. We’ll dive into the science behind this innovative approach, its development, and the promising results from clinical trials that demonstrate improved patient outcomes. Join us as we uncover how Givosiran is reshaping the landscape of AHP treatment, providing new hope for patients managing this challenging condition.
Creator: Kiera Sha
References:
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