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Orphanet Journal of Rare Diseases|August 8, 2024
Pozelimab for CHAPLE disease: results from in-trial interviews and clinical outcome assessmentsLeighann Litcher-Kelly, Ahmet Ozen, Sarah Ollis, et al.
Plos One|March 11, 2014
Impact of the Smoothened inhibitor, IPI-926, on smoothened ciliary localization and Hedgehog pathway activityMarisa O Peluso, Veronica T Campbell, Joseph A Harari, et al.
Orphanet Journal of Rare Diseases|February 11, 2025
The patient experience of CHAPLE disease: results from interviews conducted as part of a clinical trial for an ultra-rare conditionLeighann Litcher-Kelly, Ahmet Ozen, Sarah Ollis, et al.
Lancet (London, England)|January 26, 2024
Evaluating the efficacy and safety of pozelimab in patients with CD55 deficiency with hyperactivation of complement, angiopathic thrombosis, and protein-losing enteropathy disease: an open-label phase 2 and 3 studyAhmet Ozen, Voranush Chongsrisawat, Asena Pinar Sefer, et al.
Annals of Oncology : Official Journal of the European Society for Medical Oncology|May 22, 2025
Spatiotemporal T-cell tracking for personalized T-cell receptor T-cell therapy designs in childhood cancerI Sentís, J L Melero, A Cebria-Xart, et al.
JCI Insight|June 23, 2025
A TMPRSS6-inhibiting mAb improves disease in a β-thalassemia mouse model and reduces iron in healthy humansHeinrich E Lob, Nikhil Singh, Kusha Mohammadi, et al.
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