A Phase I, Randomised, First-in-Human Study of an Antisense Oligonucleotide Directed Against SOD1 Delivered Intrathecally in SOD1-Familial ALS Patients

OBJECTIVE: To evaluate the safety, tolerability, and pharmacokinetics of an antisense oligonucleotide designed to inhibit SOD1 expression (ISIS 333611) following intrathecal administration in patients with SOD1-related familial amyotrophic lateral sclerosis (ALS). BACKGROUND: Mutations in SOD1 cause...

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Bibliografski detalji
Glavni autori: Miller, Timothy, Pestronk, Alan, David, William, Rothstein, Jeffrey, Simpson, Ericka, Appel, Stanley H., Andres, Patricia L., Mahoney, Katy, Allred, Peggy, Alexander, Katie, Ostrow, Lyle W., Schoenfeld, David, Macklin, Eric A., Norris, Daniel A., Manousakis, Georgios, Crisp, Matthew, Smith, Richard, Bennett, C.F., Bishop, Kathie, Cudkowicz, Merit E
Format: Artigo
Jezik:Inglês
Izdano: 2013
Teme:
Article
Online pristup:https://ncbi.nlm.nih.gov/pmc/articles/PMC3712285/
https://ncbi.nlm.nih.gov/pubmed/23541756
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/S1474-4422(13)70061-9
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