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Last Updated: 10/31/2025
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Found 114 publications
Identification of oligonucleotide drug impurities using heart-cutting two-dimensional liquid chromatography-tandem mass spectrometry (2D-LC-MS/MS).
Journal: Analytica chimica acta
Published: May 25, 2025
The Fomivirsen, Patisiran, and Givosiran Odyssey: How the Success Stories May Pave the Way for Future Clinical Translation of Nucleic Acid Drugs.
Journal: BioDrugs : clinical immunotherapeutics, biopharmaceuticals and gene therapy
Published: February 20, 2025
Development, opportunities, and challenges of siRNA nucleic acid drugs.
Journal: Molecular therapy. Nucleic acids
Published: February 03, 2025
Successful Pregnancy After Combined Liver and Renal Transplantation in a Patient With Acute Intermittent Porphyria.
Journal: Case reports in transplantation
Published: January 08, 2025
Givosiran: a targeted treatment for acute intermittent porphyria.
Journal: Hematology. American Society of Hematology. Education Program
Published: December 07, 2024
Acute Intermittent Porphyria in an Adolescent Patient: Diagnostic and Treatment Challenges.
Journal: Cureus
Published: November 29, 2024
Baseline urinary ALA and PBG as criteria for starting pharmacologic prophylactic treatment in acute intermittent porphyria treated with givosiran.
Journal: Molecular genetics and metabolism reports
Published: October 14, 2024
Efficacy and safety of givosiran in Japanese patients with acute hepatic porphyria: clinical findings from an expanded access study.
Journal: Scientific reports
Published: October 04, 2024
Neuralgic amyotrophy presentation of acute intermittent porphyria: A case report.
Journal: Journal of the peripheral nervous system : JPNS
Published: September 17, 2024
German Real-World Experience of Patients with Diverse Features of Acute Intermittent Porphyria Treated with Givosiran.
Journal: Journal of clinical medicine
Published: September 03, 2024
Separation and Characterization of Therapeutic Oligonucleotide Isomer Impurities by Cyclic Ion Mobility Mass Spectrometry.
Journal: Journal of the American Society for Mass Spectrometry
Published: July 31, 2024
Therapeutic approach to acute crises of hepatic porphyrias.
Journal: Revista clinica espanola
Published: June 22, 2024
Last Updated: 10/31/2025