Takeda/haemophilia: blood test

a breakthrough inIf gene therapy lives up to its promise, it reallywill justify the song and dance
Source: FT.com - Drugs and Healthcare - Category: Pharmaceuticals Source Type: news

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Authors: Rodriguez-Merchan EC Abstract Introduction: The purpose of this article is to review which data about hemophilia are currently provided by the Cochrane database of systematic reviews (CDBSR). Methodological consideration: All statements about hemophilia in the Cochrane Collaboration are based on evidence generated in randomized controlled clinical trials. Areas covered: There is a high degree of evidence that prophylaxis preserves joint function in children with hemophilia compared to on-demand treatment. Also, that recombinant factor VII activated (rFVIIa) and activated prothrombin complex concentrates (A...
Source: Expert Review of Hematology - Category: Hematology Tags: Expert Rev Hematol Source Type: research
CONCLUSIONS: Our results demonstrate that the FVIII-specific sensitization and expansion protocol yields more potent Tregs to suppress anti-FVIII antibody responses and induce long term tolerance to FVIII, increasing the potential for adoptive Treg cell therapy to modulate anti-FVIII immune responses. PMID: 31609041 [PubMed - as supplied by publisher]
Source: Thrombosis and Haemostasis - Category: Hematology Authors: Tags: J Thromb Haemost Source Type: research
Abstract Novel gene therapy strategies have changed the prognosis of many inherited diseases in recent years. New development in genetic tools and study models has brought us closer to a complete cure for hemophilia. This review will address the latest gene therapy research in hemophilia A and B including gene therapy tools, genetic strategies and animal models, and summarizes results of recent clinical trials. We will also discuss potential solutions to the current barriers in gene therapy for hemophilia. PMID: 31549954 [PubMed - as supplied by publisher]
Source: Current Gene Therapy - Category: Genetics & Stem Cells Authors: Tags: Curr Gene Ther Source Type: research
This article looks at current treatment models for prophylaxis and management of inhibitors, reviews the recent advances in the field (with bioengineered factor products, non-factor products and gene therapy) and summarizes the incorporation of these new interventions in the treatment plan for patients with hemophilia.
Source: Indian Journal of Pediatrics - Category: Pediatrics Source Type: research
Abstract Trials of antigen-specific tolerance have been undertaken in the clinic for over fifty years and the results of these antigen-specific clinical trials are described in this review. Antigen-specific tolerization of the immune system in protein replacement therapy for hemophilia A is an accepted treatment. Clinical trials are ongoing for autoimmune conditions such as type 1 diabetes, multiple sclerosis, neuromyelitis optica, and rheumatoid arthritis with various antigen-specific strategies. Trials for tolerization in celiac disease aim for antigen specific tolerance to gluten, an environmental trigger, whic...
Source: Current Opinion in Immunology - Category: Allergy & Immunology Authors: Tags: Curr Opin Immunol Source Type: research
CONCLUSIONS: These improved structures yield a more confident model for next generation engineering efforts to develop FVIII therapeutics with longer half-lives, higher expression levels and lower immunogenicity. This article is protected by copyright. All rights reserved. PMID: 31454152 [PubMed - as supplied by publisher]
Source: Thrombosis and Haemostasis - Category: Hematology Authors: Tags: J Thromb Haemost Source Type: research
Authors: Kizilocak H, Young G Abstract Hemophilia A and B are inherited bleeding disorders characterized by deficiency or dysfunction of coagulation protein factors VIII and IX, respectively. Recurrent joint and muscle bleeds are the major clinical manifestations. Replacement therapy with clotting factors, either at the time of bleeding or as part of a prophylactic regimen, is adapted to individual patient needs. The major complication of therapy is the development of neutralizing antibodies. In response, researchers have developed novel agents to both reduce the treatment burden and prevent bleeding regardless of ...
Source: Clinical Advances in Hematology and Oncology - Category: Cancer & Oncology Tags: Clin Adv Hematol Oncol Source Type: research
Semin Thromb Hemost DOI: 10.1055/s-0039-1692977Clinicians and scientists in the fields of hemostasis and thrombosis have been among those first to integrate new molecular strategies for the purpose of enhancing disease diagnosis and treatment. The molecular diagnosis and introduction of gene therapy approaches for hemophilia are obvious examples of this tendency. In this review, the authors summarize information concerning three molecular technologies that have reached various stages of translational potential for their incorporation into the clinical management of disorders of hemostasis. Chromatin conformation assays are...
Source: Seminars in Thrombosis and Hemostasis - Category: Hematology Authors: Tags: Review Article Source Type: research
I am a participant in the Children ’s Hospital of Philadelphia’s (CHOP) Gene Therapy Trial for Hemophilia B (SPK 9001).
Source: Transfusion and Apheresis Science - Category: Hematology Authors: Source Type: research
Over the past decades hemophilia has been transformed from a debilitating disease to a manageable condition. However, the current treatment options are expensive, complex, and inaccessible to a large portion of the global population. Moreover, the development of antibodies to replacement factors, termed inhibitors, is a common complication that not only renders conventional prophylaxis regimens ineffective but also increase the annual bleeding rate in affected patients. Fortunately, much progress has been made toward developing a curative gene therapy treatment for hemophilia and these efforts have led to a series of human...
Source: Transfusion and Apheresis Science - Category: Hematology Authors: Source Type: research
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