The New England journal of medicine, 2020-07, Vol.383 (2), p.109-119
- Miller, Timothy
- Cudkowicz, Merit
- Shaw, Pamela J
- Andersen, Peter M
- Atassi, Nazem
- Bucelli, Robert C
- Genge, Angela
- Glass, Jonathan
- Ladha, Shafeeq
- Ludolph, Albert L
- Maragakis, Nicholas J
- McDermott, Christopher J
- Pestronk, Alan
- Ravits, John
- Salachas, François
- Trudell, Randall
- Van Damme, Philip
- Zinman, Lorne
- Bennett, C. Frank
- Lane, Roger
- Sandrock, Alfred
- Runz, Heiko
- Graham, Danielle
- Houshyar, Hani
- McCampbell, Alexander
- Nestorov, Ivan
- Chang, Ih
- McNeill, Manjit
- Fanning, Laura
- Fradette, Stephanie
- Ferguson, Toby A
2020
Details
- Autor(en) / Beteiligte
- Miller, Timothy
- Cudkowicz, Merit
- Shaw, Pamela J
- Andersen, Peter M
- Atassi, Nazem
- Bucelli, Robert C
- Genge, Angela
- Glass, Jonathan
- Ladha, Shafeeq
- Ludolph, Albert L
- Maragakis, Nicholas J
- McDermott, Christopher J
- Pestronk, Alan
- Ravits, John
- Salachas, François
- Trudell, Randall
- Van Damme, Philip
- Zinman, Lorne
- Bennett, C. Frank
- Lane, Roger
- Sandrock, Alfred
- Runz, Heiko
- Graham, Danielle
- Houshyar, Hani
- McCampbell, Alexander
- Nestorov, Ivan
- Chang, Ih
- McNeill, Manjit
- Fanning, Laura
- Fradette, Stephanie
- Ferguson, Toby A
- Titel
- Phase 1–2 Trial of Antisense Oligonucleotide Tofersen for SOD1 ALS
- Ist Teil von
- The New England journal of medicine, 2020-07, Vol.383 (2), p.109-119
- Ort / Verlag
- United States: Massachusetts Medical Society
- Erscheinungsjahr
- 2020
- Link zum Volltext
- Quelle
- Free E-Journal (出版社公開部分のみ）
- Beschreibungen/Notizen
- In a phase 1–2 dose-escalation trial involving adults with ALS due to SOD1 mutations who received intrathecal tofersen (an antisense oligonucleotide) or placebo, the levels of mutant SOD1 in the CSF were 33 percentage points lower in the highest-dose tofersen group than in the placebo group.
- Sprache
- Englisch
- Identifikatoren
- ISSN: 0028-4793, 1533-4406eISSN: 1533-4406DOI: 10.1056/NEJMoa2003715Titel-ID: cdi_swepub_primary_oai_DiVA_org_umu_175822
- Format
- –
- Schlagworte
- Adult, Amyotrophic lateral sclerosis, Amyotrophic Lateral Sclerosis - cerebrospinal fluid, Amyotrophic Lateral Sclerosis - drug therapy, Amyotrophic Lateral Sclerosis - genetics, Antisense drugs, Antisense oligonucleotides, Causes of, Cerebrospinal fluid, Complications and side effects, Disease Progression, Dose-Response Relationship, Drug, Double-Blind Method, Drug therapy, Embolism, Female, Genetic aspects, Headache - chemically induced, Health aspects, Humans, Injections, Spinal - adverse effects, Intermediate Filaments, Investigations, Leukocytosis - chemically induced, Male, Medical prognosis, Middle Aged, mRNA, Mutation, Oligonucleotides, Oligonucleotides - administration & dosage, Oligonucleotides - adverse effects, Oligonucleotides - pharmacokinetics, Oligonucleotides, Antisense - administration & dosage, Oligonucleotides, Antisense - adverse effects, Oligonucleotides, Antisense - pharmacokinetics, Oxidoreductases, Pharmacodynamics, Pharmacokinetics, Physiological aspects, Pleocytosis, Protein biosynthesis, Proteins, Respiratory failure, Statistical analysis, Superoxide dismutase, Superoxide Dismutase-1 - cerebrospinal fluid, Superoxide Dismutase-1 - genetics, Testing, Vital Capacity