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

Acute intermittent porphyria results in excess activity of ALA synthase and overproduction of neurotoxic metabolites that cause attacks of severe abdominal pain. Givosiran, an interfering RNA, blocks synthesis of the enzyme, reduces the attack rate, and has only mild-to-moderate side effects.

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Bibliographische Detailangaben
Veröffentlicht in:	The New England journal of medicine 2019-02, Vol.380 (6), p.549-558
Hauptverfasser:	Sardh, Eliane, Harper, Pauline, Balwani, Manisha, Stein, Penelope, Rees, David, Bissell, D. Montgomery, Desnick, Robert, Parker, Charles, Phillips, John, Bonkovsky, Herbert L, Vassiliou, Daphne, Penz, Craig, Chan-Daniels, Amy, He, Qiuling, Querbes, William, Fitzgerald, Kevin, Kim, Jae B, Garg, Pushkal, Vaishnaw, Akshay, Simon, Amy R, Anderson, Karl E
Format:	Artikel
Sprache:	eng
Schlagworte:	Adult Amides - administration & dosage Amides - adverse effects Aminolevulinic acid Body weight Diarrhea Dose-Response Relationship, Drug Drug Administration Schedule Female Gene expression Heme Hereditary diseases Humans Injections, Subcutaneous Intermediates Liver - metabolism Liver diseases Male Middle Aged Molecular Targeted Therapy Mutation Neurotoxicity Pain Patients Pharmaceuticals Pharmacodynamics Porphobilinogen - blood Porphyria Porphyria, Acute Intermittent - drug therapy Rhinopharyngitis RNA, Messenger - metabolism RNA, Messenger - urine RNA-mediated interference RNAi Therapeutics
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Beschreibung
Zusammenfassung:	Acute intermittent porphyria results in excess activity of ALA synthase and overproduction of neurotoxic metabolites that cause attacks of severe abdominal pain. Givosiran, an interfering RNA, blocks synthesis of the enzyme, reduces the attack rate, and has only mild-to-moderate side effects.
ISSN:	0028-4793 1533-4406
DOI:	10.1056/NEJMoa1807838