Shire Announces FDA Approval of Cuvitru immune globulin subcutaneous (human) for Primary Immunodeficiency

Lexington, Mass. – September 14, 2016 – Shire plc (LSE: SHP, NASDAQ: SHPG) announced that the United States Food and Drug Administration (FDA) has granted approval for Cuvitru [Immune Globulin Subcutaneous (Human), 20% Solution] in adult and...
Source: Drugs.com - New Drug Approvals - Category: Drugs & Pharmacology Source Type: news

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ConclusionsThe authors highlight the importance of detailed immunological investigations in an infant with severe infections and lymphopenia before labeling the infant as SCID.
Source: Indian Journal of Pediatrics - Category: Pediatrics Source Type: research
Publication date: Available online 12 March 2019Source: International Journal of Pediatrics and Adolescent MedicineAuthor(s): Yousef Almana, Reem MohammedAbstractInflammatory bowel disease (IBD) is a heterogeneous group of disorders composed mainly of Ulcerative Colitis (UC) and Crohn’s disease (CD) and Undetermined IBD. The peak incidence of occurrence is mainly beyond the pediatric age group. Recent knowledge about genetic factors had been strongly linked to pediatric IBD (PIBD). Recent advances in genomic technologies have prompted the identification of genetic defects underlying rare, very early‐onset IBD (VEO...
Source: International Journal of Pediatrics and Adolescent Medicine - Category: Pediatrics Source Type: research
AbstractX-linked chronic granulomatous disease (XL-CGD), a rare primary immunodeficiency due to a defect in the gp91phox NADPH oxidase subunit, results in recurrent, severe infection, inflammation, and autoimmunity. Patients have an absent, or significantly reduced, neutrophil oxidative burst. Due to lyonization, XL-CGD carriers have a dual population of functional and non-functional phagocytes and experience a range of symptoms including increased risk of autoimmunity, fatigue, and infection. Patients with CGD have poorer quality of life (QoL) than normal controls. We evaluated QoL and psychological health in UK XL-CGD ca...
Source: Journal of Clinical Immunology - Category: Allergy & Immunology Source Type: research
Allogenic hematopoietic stem cell transplantation (HSCT) is a treatment option for children with various malignant or non-malignant diseases, including selected primary immunodeficiencies (PIDs) and inherited disorders. A graft from a healthy, HLA-genoidentical, matched sibling donor is the best option but is available in fewer than 25% of cases. Less than 70% of the remaining patients will have a suitable matched unrelated donor, a proportion that is even lower for patients belonging to ethnic groups poorly represented in donor registries [1].
Source: Biology of Blood and Marrow Transplantation - Category: Hematology Authors: Source Type: research
We report the first case of hypomorphic X-SCID caused by a synonymous mutation in the IL2RG gene leading to a splice anomaly, in a family including two patients with diffuse cutaneous warts, recurrent molluscum contagiosum, and mild respiratory infections. The mutation caused aberrant splicing of IL2RG mRNA, subsequently resulted in reduced γc expression. The leaky production of normally spliced IL2RG mRNA produced undamaged protein; thus, T cells and NK cells were generated in the patients. Functional assays of the patients' T cells and NK cells revealed diminished cytokine response in the T cells and absent cytokin...
Source: International Journal of Hematology - Category: Hematology Authors: Tags: Int J Hematol Source Type: research
PMID: 30858051 [PubMed - as supplied by publisher]
Source: Clinical Immunology - Category: Allergy & Immunology Authors: Tags: Clin Immunol Source Type: research
To investigate effects and outcome of hematopoietic stem cell transplantation (HSCT) on sclerosing cholangitis, in pediatric patients with different primary immunodeficiencies (PIDs).
Source: The Journal of Pediatrics - Category: Pediatrics Authors: Tags: Original Articles Source Type: research
Mirjam van der Burg, Tomas Kalina, Martin Perez-Andres, Marcela Vlkova, Eduardo Lopez-Granados, Elena Blanco, Carolien Bonroy, Ana E. Sousa, Anne-Kathrin Kienzler, Marjolein Wentink, Ester Mejstr íková, Vendula Šinkorova, Jan Stuchly, Menno C. van Zelm, Alberto Orfao, Jacques J. M. van Dongen
Source: Frontiers in Immunology - Category: Allergy & Immunology Source Type: research
Publication date: March 2019Source: The Journal of Allergy and Clinical Immunology: In Practice, Volume 7, Issue 3Author(s): Evans R. Fernández Pérez, Maya Hunter, Rohit K. Katial
Source: The Journal of Allergy and Clinical Immunology: In Practice - Category: Allergy & Immunology Source Type: research
Publication date: March 2019Source: The Journal of Allergy and Clinical Immunology: In Practice, Volume 7, Issue 3Author(s): John B. Ziegler, Mark Ballow
Source: The Journal of Allergy and Clinical Immunology: In Practice - Category: Allergy & Immunology Source Type: research
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