Tominersen in Adults with Manifest Huntington's Disease

Tominersen and Huntington’s DiseaseA trial of tominersen, designed to slow Huntington’s disease progression by lowering levels of huntingtin protein, was stopped prematurely, and an ad hoc analysis of the results at week 69 was carried out.

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Bibliographische Detailangaben
Veröffentlicht in:	The New England journal of medicine 2023-12, Vol.389 (23), p.2203-2205
Hauptverfasser:	McColgan, Peter, Thobhani, Alpa, Boak, Lauren, Schobel, Scott A, Nicotra, Alessia, Palermo, Giuseppe, Trundell, Dylan, Zhou, Julian, Schlegel, Valerie, Sanwald Ducray, Patricia, Hawellek, David J, Dorn, Jonas, Simillion, Cedric, Lindemann, Michael, Wheelock, Vicki, Durr, Alexandra, Anderson, Karen E, Long, Jeffrey D, Wild, Edward J, Landwehrmeyer, G Bernhard, Leavitt, Blair R, Tabrizi, Sarah J, Doody, Rachelle
Format:	Artikel
Sprache:	eng
Schlagworte:	Adult Biomarkers Cerebrospinal fluid Humans Huntingtin Huntington Disease - diagnosis Huntington Disease - drug therapy Hypotheses Oligonucleotides - therapeutic use Proteins
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Beschreibung
Zusammenfassung:	Tominersen and Huntington’s DiseaseA trial of tominersen, designed to slow Huntington’s disease progression by lowering levels of huntingtin protein, was stopped prematurely, and an ad hoc analysis of the results at week 69 was carried out.
ISSN:	0028-4793 1533-4406
DOI:	10.1056/NEJMc2300400