The New England journal of medicine, 2022-09, Vol.387 (12), p.1099-1110
- Miller, Timothy M.
- Cudkowicz, Merit E.
- Genge, Angela
- Shaw, Pamela J.
- Sobue, Gen
- Bucelli, Robert C.
- Chiò, Adriano
- Van Damme, Philip
- Ludolph, Albert C.
- Glass, Jonathan D.
- Andrews, Jinsy A.
- Babu, Suma
- Benatar, Michael
- McDermott, Christopher J.
- Cochrane, Thos
- Chary, Sowmya
- Chew, Sheena
- Zhu, Han
- Wu, Fan
- Nestorov, Ivan
- Graham, Danielle
- Sun, Peng
- McNeill, Manjit
- Fanning, Laura
- Ferguson, Toby A.
- Fradette, Stephanie
2022
Details
- Autor(en) / Beteiligte
- Miller, Timothy M.
- Cudkowicz, Merit E.
- Genge, Angela
- Shaw, Pamela J.
- Sobue, Gen
- Bucelli, Robert C.
- Chiò, Adriano
- Van Damme, Philip
- Ludolph, Albert C.
- Glass, Jonathan D.
- Andrews, Jinsy A.
- Babu, Suma
- Benatar, Michael
- McDermott, Christopher J.
- Cochrane, Thos
- Chary, Sowmya
- Chew, Sheena
- Zhu, Han
- Wu, Fan
- Nestorov, Ivan
- Graham, Danielle
- Sun, Peng
- McNeill, Manjit
- Fanning, Laura
- Ferguson, Toby A.
- Fradette, Stephanie
- Titel
- Trial of Antisense Oligonucleotide Tofersen for SOD1 ALS
- Ist Teil von
- The New England journal of medicine, 2022-09, Vol.387 (12), p.1099-1110
- Ort / Verlag
- Boston: Massachusetts Medical Society
- Erscheinungsjahr
- 2022
- Link zum Volltext
- Quelle
- Alma/SFX Local Collection
- Beschreibungen/Notizen
- The antisense molecule tofersen was tested intrathecally in 108 patients with ALS due to SOD1 mutations. In a subgroup with faster-progressing disease, there was no clinical difference from placebo over a period of 28 weeks.
- Sprache
- Englisch
- Identifikatoren
- ISSN: 0028-4793eISSN: 1533-4406DOI: 10.1056/NEJMoa2204705Titel-ID: cdi_proquest_miscellaneous_2716932393
- Format
- –
- Schlagworte
- Adverse events, Amyotrophic lateral sclerosis, Antisense drugs, Antisense oligonucleotides, Antisense therapy, Cerebrospinal fluid, Drug therapy, Genetics, Genetics General, Health aspects, Light chains, Muscles, Mutation, Neurology, Neuromuscular Disease, Neurosurgery, Oligonucleotides, Patient outcomes, Placebos, Proteins, Questionnaires, Superoxide dismutase, Testing, Ventilation