WHIM syndrome


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WHIM syndrome

acronym for genetic syndrome [MIM 193670] comprising warts, hypogammaglobulinemia, infections, and myelokathexis; autosomal dominant.

WHIM syndrome

acronym for genetic syndrome [MIM 193670] comprising warts, hypogammaglobulinemia, infections, and myelokathexis; autosomal dominant.
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X4's most advanced product candidate, mavorixafor (X4P-001), is in a global Phase 3 pivotal trial in patients with WHIM syndrome, a rare, inherited, primary immunodeficiency disease, and is currently also under investigation in combination with axitinib in the Phase 2a portion of an open-label Phase 1/2 clinical trial in clear cell renal cell carcinoma (ccRCC).
M2 PHARMA-July 31, 2019-X4 Pharmaceuticals Receives Orphan Drug Designation from European Commission for Mavorixafor for Treatment of WHIM Syndrome
WHIM syndrome is a primary immunodeficiency disorder characterized by panleukopenia and caused by autosomal dominant gain-of-function mutations in CXC chemokine receptor 4 (CXCR4).
The company added that its most advanced product candidate is in a Phase 2/3 clinical trial in patients with WHIM syndrome, a rare genetic, primary immunodeficiency disease and is currently under investigation in multiple clinical trials in solid tumours.
The woman had been diagnosed as a child with WHIM syndrome, an immune disease caused by a mutation in a gene called CXCR4.
Chromothriptic cure of WHIM syndrome. Cell 2015 Feb;160(4):686-699.
WHIM Syndrome. Warts, hypogammaglobulinemia, infections, and myelokathexis (WHIM) is an immunodeficiency with autosomal dominant inheritance.
The company's most advanced product candidate, mavorixafor (X4P-001), is expected to commence a global Phase 3 pivotal trial in patients with WHIM syndrome, a rare genetic, primary immunodeficiency disease, in 2Q19 and is currently also under investigation in a Phase 2a clinical trial in clear cell renal cell carcinoma.
United States-based X4 Pharmaceuticals' X4P-001-RD has demonstrated promising activity in the ongoing open-label phase two portion of a phase 2/3 study in patients with WHIM syndrome, a primary immunodeficiency disease caused by a mutation in the C-X-C receptor type 4 (CXCR4) gene, which results in susceptibility to certain types of infections, it was reported yesterday.
People with a rare disorder known as WHIM syndrome suffer warts and recurrent bacterial infections.
ENPNewswire-July 31, 2019--X4 Pharmaceuticals Receives Orphan Drug Designation from European Commission for Mavorixafor for Treatment of WHIM Syndrome
M2 PHARMA-December 4, 2018-X4 Pharmaceuticals Presents Additional Positive Phase 2 Results for X4P-001 in WHIM Syndrome, Continues on Path to Initiation of Phase 3 Trial