Written by Jennifer, a patient living with WHIM syndrome. ### I’m in my sixties - but it wasn’t until a decade ago that I learned the name of the rare…
In the past, treatment for WHIM syndrome has focused on symptom management: treating infections and myelokathexis with antibiotics or granulocyte colony stimulating factor. Infusion can treat hypogammaglobulinemia and surgery…
Welcome to the Rare Classroom, a new series from Patient Worthy. Rare Classroom is designed for the curious reader who wants to get informed on some of the rarest, most…
X4 Pharmaceuticals Inc. has recently published data from 3 different posters which were presented at the American Society of Hematology (ASH) Annual Meeting in Georgia. You can read the full…
Clinical trial recruitment has always been challenging. It is especially challenging for therapies being tested for rare diseases, an already small population. Clinical trials for rare diseases are small out…
According to a story from GlobeNewswire, the biopharmaceutical company X4 Pharmaceuticals, Inc. has just announced that the US Food and Drug Administration (FDA) has granted its investigational product candidate mavorixafor…
WHIM Syndrome WHIM syndrome is a form of primary immunodeficiency. It is caused by a mutation in the CXCR4 gene. WHIM stands for Warts, Hypogammaglobulinemia, Infections, and Myelokathexis. Throughout their…
According to a story from home.suddenlink.net, the biopharmaceutical company X4 Pharmaceuticals recently announced that the European Commission has granted Orphan Drug designation to the company's lead investigational drug candidate, mavorixafor.…
According to a story from BioPortfolio, the biopharmaceutical company X4 Pharmaceuticals, Inc. has recently initiated a phase 3 clinical trial that is testing its current lead product candidate mavorixafor. This…
According to a story from Business Wire, the biopharmaceutical company X4 Pharmaceuticals, Inc. and the medical genetics company Invitae Corporation have recently announced that they have come into a parternship…
According to an article published by Biospace, X4 Pharmaceuticals, based in Cambridge, Massachusetts, has recently announced successful phase two results in clinical trials for their experimental drug X4P-001. The drug…
According to a story from BioSpace, the biotechnology company X4 Pharmaceuticals recently announced that the US Food and Drug Administration (FDA) has given Orphan Drug designation to the company's investigational…
Clinical data from a Phase 2 study of the experimental drug X4P-001-RD for the treatment of WHIM syndrome has been announced at the European Hematology Association’s 23rdCongress in Stockholm. The…
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