Pharmacokinetic‐pharmacodynamic model of urinary δ‐aminolevulinic acid reduction after givosiran treatment in patients with acute hepatic porphyria

• मुख्य लेखकों: Jongtae Lee (लेखक), Megan Melch (लेखक), Gabriel J. Robbie (लेखक)
• स्वरूप: पुस्तक
• प्रकाशित: Wiley, 2023-06-01T00:00:00Z.
• विषय: article
• ऑनलाइन पहुंच: Connect to this object online.

Abstract Givosiran, an RNA interference‐based therapeutic, is a recent addition to the limited treatment armamentarium for acute hepatic porphyria (AHP). As a small interfering RNA that is selectively taken up in the liver, both the mechanism and targeted delivery create a complex relationship betwe...