Nirogacestat, a γ-secretase inhibitor for desmoid tumors
In a randomized, placebo-controlled trial, oral nirogacestat twice daily led to 41% of patients having a tumor response, and 2-year progression-free survival was 76%. Most adverse events were low grade.
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| Hauptverfasser: | , , , , , , , , , , , , , , , , , , , , , , , , , , , , , , , , , , , , , , , , |
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| Dokumenttyp: | Article (Journal) |
| Sprache: | Englisch |
| Veröffentlicht: |
March 09, 2023
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| In: |
The New England journal of medicine
Year: 2023, Jahrgang: 388, Heft: 10, Pages: 898-912 |
| ISSN: | 1533-4406 |
| DOI: | 10.1056/NEJMoa2210140 |
| Online-Zugang: | Verlag, lizenzpflichtig, Volltext: https://doi.org/10.1056/NEJMoa2210140 Verlag, lizenzpflichtig, Volltext: https://www.nejm.org/doi/full/10.1056/NEJMoa2210140 |
| Verfasserangaben: | Mrinal Gounder, Ravin Ratan, Thierry Alcindor, Patrick Schöffski, Winette T. van der Graaf, Breelyn A. Wilky, Richard F. Riedel, Allison Lim, L. Mary Smith, Stephanie Moody, Steven Attia, Sant Chawla, Gina D’Amato, Noah Federman, Priscilla Merriam, Brian A. Van Tine, Bruno Vincenzi, Charlotte Benson, Nam Quoc Bui, Rashmi Chugh, Gabriel Tinoco, John Charlson, Palma Dileo, Lee Hartner, Lore Lapeire, Filomena Mazzeo, Emanuela Palmerini, Peter Reichardt, Silvia Stacchiotti, Howard H. Bailey, Melissa A. Burgess, Gregory M. Cote, Lara E. Davis, Hari Deshpande, Hans Gelderblom, Giovanni Grignani, Elizabeth Loggers, Tony Philip, Joseph G. Pressey, Shivaani Kummar, and Bernd Kasper |
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| 520 | |a In a randomized, placebo-controlled trial, oral nirogacestat twice daily led to 41% of patients having a tumor response, and 2-year progression-free survival was 76%. Most adverse events were low grade. | ||
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