Results of a phase 2 trial of an oral CXCR4 antagonist, mavorixafor, for treatment of WHIM syndrome

Warts, hypogammaglobulinemia, infections, and myelokathexis (WHIM) syndrome is a rare primary immunodeficiency caused by gain-of-function mutations in the CXCR4 gene. We report the safety, tolerability, pharmacokinetics, pharmacodynamics, and preliminary efficacy of mavorixafor from a phase 2 open-l...

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Kaydedildi:
Detaylı Bibliyografya
Yayımlandı:Blood
Asıl Yazarlar: Dale, David C., Firkin, Frank, Bolyard, Audrey Anna, Kelley, Merideth, Makaryan, Vahagn, Gorelick, Kenneth J., Ebrahim, Tarek, Garg, Varun, Tang, Weihua, Jiang, Honghua, Skerlj, Renato, Beaussant Cohen, Sarah
Materyal Türü: Artigo
Dil:Inglês
Baskı/Yayın Bilgisi: American Society of Hematology 2020
Konular:
Clinical Trials and Observations
Online Erişim:https://ncbi.nlm.nih.gov/pmc/articles/PMC7770568/
https://ncbi.nlm.nih.gov/pubmed/32870250
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1182/blood.2020007197
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