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Givosiran for the Treatment of Pediatric Acute Intermittent Porphyria

Kenneth E Bujold1, Nicole Kasher2, Christine McKiernan3

  • 1Division of Pediatric Hematology-Oncology, Baystate Children's Hospital.

Journal of Pediatric Hematology/Oncology
|August 23, 2024
PubMed

Insights

Givosiran therapy offers a potential new treatment for pediatric acute intermittent porphyria (AIP), significantly improving a patient

Area of Science:

  • Biochemistry
  • Genetics
  • Pediatric Medicine

Background:

  • Acute intermittent porphyria (AIP) is a rare genetic disorder.
  • AIP causes severe neurovisceral symptoms and organ damage.
  • Current treatments for AIP include trigger avoidance and hemin infusions.

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