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Wiskott-Aldrich Syndrome

A rare, X-linked immunodeficiency syndrome characterized by ECZEMA; LYMPHOPENIA; and, recurrent pyogenic infection. It is seen exclusively in young boys. Typically, IMMUNOGLOBULIN M levels are low and IMMUNOGLOBULIN A and IMMUNOGLOBULIN E levels are elevated. Lymphoreticular malignancies are common.
Also Known As:
Eczema-Thrombocytopenia-Immunodeficiency Syndrome; Imd2; Immunodeficiency 2; Wiskott Syndrome; Eczema Thrombocytopenia Immunodeficiency Syndrome; Eczema-Thrombocytopenia-Immunodeficiency Syndromes; Immunodeficiency 2s; Wiskott Aldrich Syndrome; Wiskott Syndromes; Aldrich Syndrome
Networked: 710 relevant articles (24 outcomes, 37 trials/studies)

Relationship Network

Disease Context: Research Results

Related Diseases

1. Thrombocytopenia (Thrombopenia)
2. Severe Combined Immunodeficiency (Bare Lymphocyte Syndrome)
3. Chronic Granulomatous Disease
4. Inborn Genetic Diseases (Disease, Hereditary)
5. Thalassemia

Experts

1. Thrasher, Adrian J: 21 articles (01/2021 - 11/2004)
2. Notarangelo, Luigi D: 18 articles (01/2020 - 07/2005)
3. Ochs, Hans D: 17 articles (01/2022 - 12/2003)
4. Aiuti, Alessandro: 16 articles (01/2022 - 08/2002)
5. Candotti, Fabio: 13 articles (10/2016 - 05/2003)
6. Geha, Raif S: 12 articles (10/2016 - 08/2002)
7. Morio, Tomohiro: 10 articles (09/2019 - 01/2004)
8. Villa, Anna: 8 articles (05/2019 - 04/2009)
9. Fischer, Alain: 7 articles (01/2022 - 10/2002)
10. Imai, Kohsuke: 7 articles (09/2019 - 12/2003)

Drugs and Biologics

Drugs and Important Biological Agents (IBA) related to Wiskott-Aldrich Syndrome:
1. Proteins (Proteins, Gene)FDA Link
2. Transfer Factor (Factor, Transfer)IBA
3. Adenosine DeaminaseIBA
06/01/2014 - "This review details gene therapy trials for X-linked and adenosine deaminase (ADA)-deficient severe combined immunodeficiency (SCID), Wiskott-Aldrich syndrome (WAS) and chronic granulomatous disease (CGD). "
04/01/2015 - "First clinical trials performed with gamma retroviral vectors (γ-RV) for adenosine deaminase severe combined immunodeficiency (ADA-SCID), X-linked SCID (SCID-X1), and Wiskott-Aldrich syndrome (WAS) showed that gene therapy is a valid therapeutic option in patients lacking an HLA-identical donor. "
01/01/2021 - "Gene therapy has successfully treated several PIDs including Adenosine Deaminase Severe Combined Immunodeficiency (SCID), X-linked SCID, Artemis SCID, Wiskott-Aldrich Syndrome, X-linked Chronic Granulomatous Disease and Leukocyte Adhesion Deficiency-I. In all, gene therapy for PIDs has progressed over the recent decades to be equal or better than allogeneic HSCT in terms of efficacy and safety. "
12/01/2019 - "Fifty-seven paediatric patients (age 0.5-11.4 years) underwent a bone marrow harvest for the purpose of HSPC gene therapy (GT), including adenosine deaminase-severe combined immunodeficiency (ADA-SCID), Wiskott-Aldrich syndrome (WAS) and metachromatic leukodystrophy (MLD) patients. "
06/13/2020 - "Recent clinical trials using patient's own corrected hematopoietic stem cells (HSCs), such as for primary immunodeficiencies (Adenosine deaminase (ADA) deficiency, X-linked Severe Combined Immunodeficiency (SCID), X-linked chronic granulomatous disease (CGD), Wiskott-Aldrich Syndrome (WAS)), have yielded promising results in the clinic; endorsing gene therapy to become standard therapy for a number of diseases. "
4. AntibodiesIBA
5. Cyclophosphamide (Cytoxan)FDA LinkGeneric
6. Busulfan (Busulfex)FDA Link
7. HLA Antigens (Human Leukocyte Antigens)IBA
8. Membrane Glycoproteins (Membrane Glycoprotein)IBA
9. Complementary DNA (cDNA)IBA
10. Adrenal Cortex Hormones (Corticosteroids)IBA

Therapies and Procedures

1. Transplantation
2. Therapeutics
3. Bone Marrow Transplantation (Transplantation, Bone Marrow)
4. Splenectomy
5. Hematopoietic Stem Cell Transplantation