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

Eliane Sardh | Zendy; Pauline Harper | Zendy; Manisha Balwani | Zendy; Penelope E. Stein | Zendy; David C. Rees | Zendy; D. Montgomery Bissell | Zendy; Robert J. Desnick | Zendy; Charles Parker | Zendy; John D. Phillips | Zendy; Herbert L. Bonkovsky | Zendy; Daphne Vassiliou | Zendy; Craig Penz | Zendy; Amy Chan-Daniels | Zendy; Qiuling He | Zendy; William Querbes | Zendy; Kevin Fitzgerald | Zendy; Jae Bum Kim | Zendy; Pushkal Garg | Zendy; Akshay Vaishnaw | Zendy; Amy Simon | Zendy; Karl E. Anderson | Zendy

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

Author(s) -

Eliane Sardh,

Pauline Harper,

Manisha Balwani,

Penelope E. Stein,

David C. Rees,

D. Montgomery Bissell,

Robert J. Desnick,

Charles Parker,

John D. Phillips,

Herbert L. Bonkovsky,

Daphne Vassiliou,

Craig Penz,

Amy Chan-Daniels,

Qiuling He,

William Querbes,

Kevin Fitzgerald,

Jae Bum Kim,

Pushkal Garg,

Akshay Vaishnaw,

Amy Simon,

Karl E. Anderson

Publication year - 2019

Publication title -

new england journal of medicine

Language(s) - English

Resource type - Journals

SCImago Journal Rank - 19.889

H-Index - 1030

eISSN - 1533-4406

pISSN - 0028-4793

DOI - 10.1056/nejmoa1807838

Subject(s) - porphyria , acute intermittent porphyria , medicine , rna interference , rna , biology , genetics , gene

Induction of delta aminolevulinic acid synthase 1 ( ALAS1) gene expression and accumulation of neurotoxic intermediates result in neurovisceral attacks and disease manifestations in patients with acute intermittent porphyria, a rare inherited disease of heme biosynthesis. Givosiran is an investigational RNA interference therapeutic agent that inhibits hepatic ALAS1 synthesis.

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