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February 6, 2019New England Journal of MedicineOpen Access

Phase 1 Trial of an RNA Interference Therapy for Acute Intermittent Porphyria

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Authors

ESEliane SardhPHPauline HarperMBManisha Balwani

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Overview

Randomized trial shows lower attack rates in acute intermittent porphyria with givosiran therapy, suggesting an effective treatment.

Key Points

  • The aim is to evaluate the safety and efficacy of givosiran in reducing porphyria attacks.
  • Phase 1 trial design with once-monthly injections of givosiran
  • Participants had recurrent porphyria attacks
  • ClinicalTrials.gov number NCT02452372
  • Givosiran treatment resulted in low-grade adverse events
  • Induced ALAS1 mRNA levels were reduced significantly
  • The attack rate was lower compared to placebo group

Cite This Study

Sardh et al. (2019) studied this question.

synapsesocial.com/papers/69dc9928a5c75be4cfe53289https://doi.org/10.1056/nejmoa1807838
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