Homo sapiens
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PROVIDER: PRJNA392090 | ENA |
REPOSITORIES: ENA
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Project description:A Prospective Natural History Study of Diagnosis, Treatment and Outcomes of Children with SCID Disorders - PIDTC 6901
| PRJNA392089 | ENA
Project description:<p>Individuals, including infants who were identified by newborn screening, with a recent/new diagnosis of severe combined immune deficiency (SCID) may be eligible to be enrolled in the PIDTC research study: A Prospective Natural History Study of Diagnosis, Treatment and Outcomes of Children with SCID Disorders. To determine eligibility for this study, potential participants (or, in the case of children, their parents/guardians) should consult with their doctors. The study follows participants with SCID prospectively. That is, the study enrolls participants who already have a plan to receive a blood and marrow transplant (BMT), enzyme therapy, or gene therapy in the future. After the procedure, participants are followed according to a study schedule. The participants' study visits will coincide with the follow-up visits with their doctors as part of their regular ongoing medical care. Participants with “leaky SCID”, reticular dysgenesis, and Omenn syndrome may also be eligible to participate in this study. As the PIDTC recognizes that there are many complex factors that go into the decision of which therapy (BMT, enzyme therapy, or gene therapy), the study does NOT dictate how the doctors should treat their patients. The decisions regarding therapy are made by the participants and their doctors. The study simply follows the participants' progress over time. This study does not involve experimental therapies.</p> <p>A Prospective Natural History Study of Diagnosis, Treatment and Outcomes of Children with SCID Disorders has been open since August 2010 and continues to be open and enrolling patients to the present day. The study plans to enroll approximately 540 patients with SCID. By studying new patients undergoing treatment for SCID, the goal is to learn more about: (1) outcomes from the treatment of SCID in the modern era of medicine, (2) what factors lead to the best long-term outcomes, such as best donor, conditioning regimen, timing of transplant, etc. and (3) what impact newborn screening and the early diagnosis of SCID has had on the long-term outcomes following BMT or gene therapy. A significant amount of information is also being gathered on how and when the immune system recovers after BMT, quality of life for long-term survivors, and about whether children develop normally after treatment. </p> <p>A Prospective Natural History Study of Diagnosis, Treatment and Outcomes of Children with SCID Disorders is the largest coordinated prospective study of patients with SCID ever performed. Information that we will learn, both now and in the future, will help doctors and other health professionals to better treat children with SCID. All hospitals within the PIDTC are enrolling patients with SCID for A Prospective Natural History Study of Diagnosis, Treatment and Outcomes of Children with SCID Disorders study, ensuring that the outcomes are reflective of what happens in the “real world” as opposed to at just one or two large centers. </p>
| phs001392 | dbGaP
Project description:Biliary atresia (BA) causes neonatal cholestasis jaundice. The primary therapeutic treatment for BA is the Kasai portoenterostomy, a surgical procedure creating a new bile duct to restore bile flow. However, current diagnostic approaches for BA are imprecise and time-consuming, making early diagnosis crucial for successful treatment outcomes. The present study was aimed to analyze proteins from peripheral blood mononuclear cells (PBMCs) obtained from children with biliary atresia compared with healthy children as potential biomarkers for diagnostic tests.
2023-10-14 | PXD043181 | JPOST Repository
Project description:Children with oligoarticular JIA (arthritis in 4 or fewer joints) can either continue to have this mild form of arthritis (persistent oligoarticular JIA) or extend to a more sever form involving more than 4 joints (extended oligoarticular JIA) Synovial fluid mononuclear cell RNA was prepared from 21 recently diagnosed pre-treatment patients and grouped according to whether the patient had extended or persisted at one year after diagnosis Each sample was hybridized to Affymetrix HGU133plus2 microarrays. The mean for each probeset was compared between groups in order to find differentially expressed genes whose expression might predict outcome in a prospective study
2009-03-04 | E-GEOD-15083 | biostudies-arrayexpress
Project description:tic disorders (TDs) are a series of childhood neuropsychiatric disorders characterized by invol-untary motor and/or vocal tics and commonly comorbid with several other psychopathological and/or behavioral disorders. In the present study, we aimed to identify serum small extracellular vesicles (sEVs) derived microRNAs (miRNAs) from the serum samples of children for Tic Disorders Diagnosis
2024-07-15 | GSE208318 | GEO
Project description:Gene expression data from whole-blood collected from Kenyan children with Plasmodium falciparum malaria infection at acute hospital admission (n=15) and at convalescence (n=9). A clinical history design type is where the organisms clinical history of diagnosis, treatments, e.g. vaccinations, surgery etc. Disease State: with Plasmodium falciparum malaria infection at acute hospital admission and at convalescence clinical_history_design
2016-02-15 | E-GEOD-35860 | biostudies-arrayexpress
Project description:Gene expression data from whole-blood collected from Kenyan children with Plasmodium falciparum malaria infection at acute hospital admission (n=15) and at convalescence (n=9). A clinical history design type is where the organisms clinical history of diagnosis, treatments, e.g. vaccinations, surgery etc. Disease State: with Plasmodium falciparum malaria infection at acute hospital admission and at convalescence
2016-02-15 | GSE35860 | GEO
Project description:To unravel genes and molecular pathways involved in the pathogenesis of type 1 diabetes (T1D), we performed genome-wide gene expression profiling of prospective venous blood samples from children developing T1D-associated autoantibodies or progressing towards clinical diagnosis.
2014-02-20 | GSE43488 | GEO
Project description:To unravel genes and molecular pathways involved in the pathogenesis of type 1 diabetes (T1D), we performed genome-wide gene expression profiling of prospective venous blood samples from children developing T1D-associated autoantibodies or progressing towards clinical diagnosis.
2014-02-20 | GSE30210 | GEO
Project description:To unravel genes and molecular pathways involved in the pathogenesis of type 1 diabetes (T1D), we performed genome-wide gene expression profiling of prospective venous blood samples from children developing T1D-associated autoantibodies or progressing towards clinical diagnosis.
2014-02-20 | GSE30209 | GEO