The New England journal of medicine
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Comment Letter
Dupilumab in Patients with Eosinophilic Esophagitis. Reply.
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Randomized Controlled Trial
Nirogacestat, a γ-Secretase Inhibitor for Desmoid Tumors.
Desmoid tumors are rare, locally aggressive, highly recurrent soft-tissue tumors without approved treatments. ⋯ Nirogacestat was associated with significant benefits with respect to progression-free survival, objective response, pain, symptom burden, physical functioning, role functioning, and health-related quality of life in adults with progressing desmoid tumors. Adverse events with nirogacestat were frequent but mostly low grade. (Funded by SpringWorks Therapeutics; DeFi ClinicalTrials.gov number, NCT03785964.).