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Familial Platelet Disorder
Clinical trial pipeline · Data from ClinicalTrials.gov
See which Familial Platelet Disorder trials you may qualify forClinical trial pipeline · Data from ClinicalTrials.gov
See which Familial Platelet Disorder trials you may qualify forBackground: Genes tell the body and its cells how to work. Familial platelet disease (FPD) or FPD with associated malignancies (FPDMM) is caused by a variant i…
Background: Runt-related transcription factor 1 (RUNX1) gene regulates the formation of blood cells. People with mutations of this gene may bleed or bruise eas…
To learn about the safety and effects of low-dose sirolimus in participants with RUNX1-FPD.
Our study is designed to characterize the clinical picture and genetic pattern of Polycythemia and Thrombocytosis. The purpose of this project is to find a gene…
To evaluate the safety and feasibility of collecting hematopoietic stem cells (HSC) in participants with RUNX1-FPD.
The goal of this clinical trial is to learn if Platelet-Rich Plasma Injections from Cord Blood (CB-PRP) is useful in Peyronie disease. The primary goal is eval…
Philadelphia-negative myeloproliferative neoplasms (MPNs) occur sporadically and are due to somatic mutations in the JAK2 (Janus kinase 2), CALR (calreticulin)…
Early Check provides voluntary screening of newborns for a selected panel of conditions. The study has three main objectives: 1) develop and implement an approa…