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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Autors principals: 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
Idioma:Inglês
Publicat: 2013
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Article
Accés en línia: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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