Targeting Huntingtin Expression in Patients with Huntington’s Disease | Zendy

Sarah J. Tabrizi | Zendy; Blair R. Leavitt | Zendy; G. Bernhard Landwehrmeyer | Zendy; Edward J. Wild | Zendy; Carsten Saft | Zendy; Roger A. Barker | Zendy; Nick F. Blair | Zendy; David Craufurd | Zendy; Josef Priller | Zendy; Hugh Rickards | Zendy; Anne Rosser | Zendy; Holly Kordasiewicz | Zendy; Christian Czech | Zendy; Eric E. Swayze | Zendy; Daniel A. Norris | Zendy; Tiffany Baumann | Zendy; Irene Gerlach | Zendy; Scott Schobel | Zendy; Erika Paz | Zendy; Anne Smith | Zendy; C. Frank Bennett | Zendy; Roger Lane | Zendy

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Targeting Huntingtin Expression in Patients with Huntington’s Disease

Author(s) -

Sarah J. Tabrizi,

Blair R. Leavitt,

G. Bernhard Landwehrmeyer,

Edward J. Wild,

Carsten Saft,

Roger A. Barker,

Nick F. Blair,

David Craufurd,

Josef Priller,

Hugh Rickards,

Anne Rosser,

Holly Kordasiewicz,

Christian Czech,

Eric E. Swayze,

Daniel A. Norris,

Tiffany Baumann,

Irene Gerlach,

Scott Schobel,

Erika Paz,

Anne Smith,

C. Frank Bennett,

Roger Lane

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/nejmoa1900907

Subject(s) - huntingtin , placebo , adverse effect , medicine , clinical endpoint , huntington's disease , pharmacokinetics , huntingtin protein , pharmacology , trinucleotide repeat expansion , dose , randomized controlled trial , disease , biology , allele , pathology , genetics , alternative medicine , gene

Huntington's disease is an autosomal-dominant neurodegenerative disease caused by CAG trinucleotide repeat expansion in HTT , resulting in a mutant huntingtin protein. IONIS-HTT Rx (hereafter, HTT Rx ) is an antisense oligonucleotide designed to inhibi HTT messenger RNA and thereby reduce concentrations of mutant huntingtin.

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