Switching from inotersen to eplontersen in patients with hereditary transthyretin-mediated amyloidosis with polyneuropathy: analysis from NEURO-TTRansform
The phase 3 NEURO-TTRansform trial showed eplontersen treatment for 65 weeks reduced transthyretin (TTR), halted progression of neuropathy impairment, and improved quality of life (QoL) in adult patients with hereditary TTR-mediated amyloidosis with polyneuropathy (ATTRv-PN), vs. historical placebo.
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| Main Authors: | , , , , , , , , , , , , , , , , |
|---|---|
| Format: | Article (Journal) |
| Language: | English |
| Published: |
13 August 2024
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| In: |
Journal of neurology
Year: 2024, Volume: 271, Issue: 10, Pages: 6655-6666 |
| ISSN: | 1432-1459 |
| DOI: | 10.1007/s00415-024-12616-6 |
| Online Access: | Verlag, kostenfrei, Volltext: https://doi.org/10.1007/s00415-024-12616-6 |
| Author Notes: | Isabel Conceição, John L. Berk, Markus Weiler, Pedro A. Kowacs, Noel R. Dasgupta, Sami Khella, Chi-Chao Chao, Shahram Attarian, T. Jesse Kwoh, Shiangtung W. Jung, Jersey Chen, Nicholas J. Viney, Rosie Z. Yu, Morie Gertz, Ahmad Masri, Márcia Waddington Cruz, Teresa Coelho |
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| 520 | |a The phase 3 NEURO-TTRansform trial showed eplontersen treatment for 65 weeks reduced transthyretin (TTR), halted progression of neuropathy impairment, and improved quality of life (QoL) in adult patients with hereditary TTR-mediated amyloidosis with polyneuropathy (ATTRv-PN), vs. historical placebo. | ||
| 650 | 4 | |a Amyloidosis | |
| 650 | 4 | |a Amyotrophic lateral sclerosis | |
| 650 | 4 | |a Antisense oligonucleotide | |
| 650 | 4 | |a Motor neuron disease | |
| 650 | 4 | |a Neurological Disorders | |
| 650 | 4 | |a Oncostatin M | |
| 650 | 4 | |a Therapeutics | |
| 650 | 4 | |a Transdifferentiation | |
| 650 | 4 | |a Transthyretin | |
| 650 | 4 | |a Treatment efficacy | |
| 650 | 4 | |a Treatment safety | |
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