Autor: |
Robert C. Bucelli, Manjit McNeill, Shafeeq Ladha, Angela Genge, Stephanie Fradette, Ivan Nestorov, John Ravits, Philip Van Damme, Roger Lane, Christopher J McDermott, Jonathan Glass, Merit Cudkowicz, Laura Fanning, C. Frank Bennett, Heiko Runz, Ih Chang, Hani Houshyar, Toby A. Ferguson, Alfred Sandrock, Alan Pestronk, Danielle Graham, Timothy M. Miller, Randall G. Trudell, Nazem Atassi, Peter M. Andersen, François Salachas, Nicholas J. Maragakis, Albert L. Ludolph, Pamela J. Shaw, Lorne Zinman, Alexander McCampbell |
Rok vydání: |
2020 |
Předmět: |
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Zdroj: |
New England Journal of Medicine. 383:109-119 |
ISSN: |
1533-4406 0028-4793 |
Popis: |
Background Tofersen is an antisense oligonucleotide that mediates the degradation of superoxide dismutase 1 (SOD1) messenger RNA to reduce SOD1 protein synthesis. Intrathecal administratio... |
Databáze: |
OpenAIRE |
Externí odkaz: |
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