Gene Therapy May Be Long-Term Cure for Type of Hemophilia

Title: Gene Therapy May Be Long-Term Cure for Type of HemophiliaCategory: Health NewsCreated: 1/2/2020 12:00:00 AMLast Editorial Review: 1/3/2020 12:00:00 AM
Source: MedicineNet Chronic Pain General - Category: Anesthesiology Source Type: news

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Conclusions: Valoctocogene roxaparvovec was found to be cost-saving-on average by about $6.8 million per patient-and more effective than prophylactic therapy for treatment of hemophilia A. The comparative benefit of gene therapy was observed across a broad range of simulated patients that were representative of the real-world severe hemophilia A population. PMID: 31971453 [PubMed - as supplied by publisher]
Source: Journal of Medical Economics - Category: Health Management Tags: J Med Econ Source Type: research
Abstract Haemophilia is a blood clotting disorder known as 'Christmas disease' caused when the blood has defect with the clotting factor(s). Bleeding leads various issues, such as chronic pain, arthritis and a serious complication during the surgery. Identifying this disease is mandatory to take the necessary treatment and maintains the normal clotting. It has been proved that the level of factor IX (FIX) is lesser with haemophilia patient and the attempt here is focused to quantify FIX level by interdigitated electrode (IDE) sensor. Single-walled carbon nanotube (SWCNT) was utilized to modify IDE sensing surface....
Source: International Journal of Biological Macromolecules - Category: Biochemistry Authors: Tags: Int J Biol Macromol Source Type: research
Conclusions:This study provides an estimate of the burden of inhibitors in persons with severe hemophilia A in the US, representing approximately 52.9% of all severe hemophilia A patients treated in the USHTCN (CDC, unpublished data). History of an inhibitor reduced patient productivity and increased ED and hospital utilization. Future efforts will focus on a longitudinal analysis of this cohort to better understand the natural history and outcome of inhibitors and their impact on patient quality of life and health care utilization.Acknowledgements:This study was performed with the advice of the Community Counts Inhibitor ...
Source: Blood - Category: Hematology Authors: Tags: 322. Disorders of Coagulation or Fibrinolysis: Poster II Source Type: research
Introduction: Recombinant Factors VIII Fc (rFVIIIFc) and IX Fc (rFIXFc) fusion proteins are extended half-life (EHL) factor products approved in Canada in 2014 and became available through the Canadian Blood Services in January 2016. Real-world product utilization and clinician practice patterns in EHL prescription are unknown.Objectives: This is a prospective Canadian multi-centre observational study to describe product utilization, clinical and patient-reported outcomes (PROs) in patients pre- and post-switch to rFVIIIFc/ rFIXFc products compared to those who remained on standard half-life (SHL) products. The primary out...
Source: Blood - Category: Hematology Authors: Tags: 903. Outcomes Research-Non-Malignant Hematology: Poster III Source Type: research
Conclusion:The 7 PROMIS domains were sensitive to several adult hemophilia disease severity indicators in a non-acute care setting. Overall scores reported by patients with hemophilia generally mirrored the normative PROMIS sample scores. However, patients with hemophilia who experienced pain reported scores significantly worse than the normative sample. PROMIS instruments provide a potentially valuable tool to study the impact of hemophilia and suggest usefulness in research and clinical practice. Additional studies are needed to assess responsiveness in PROMIS score with changes in disease status over time and in women w...
Source: Blood - Category: Hematology Authors: Tags: 903. Outcomes Research-Non-Malignant Hematology: Health Outcomes in Hemophilia, Thrombosis, ITP, and Hereditary Transthyreitin Amyloidosis Source Type: research
ConclusionsThis study underlines the value that the French, Italian, German, Swedish, United States and UK populations ascribe to the avoidance of disease progression in haemophilia without inhibitors. Improved treatment options hold a potential for important benefits to haemophilia patients.
Source: Haemophilia - Category: Hematology Authors: Tags: ORIGINAL ARTICLE Source Type: research
Source: Cardiovascular and Haematological Disorders - Drug Targets - Category: Cardiology Authors: Tags: Review article Source Type: research
Abstract BACKGROUND: Children and adolescents with severe hemophilia commonly suffer from acute and chronic pain as a consequence of hemophilia-related bleeding. Intervention-related pain also plays a major role. Despite its high prevalence in this patient group, hemophilia-related pain is not always adequately addressed and sufficiently treated. OBJECTIVES: This paper discusses how to improve pain management for children and adolescents (0-18 years) with hemophilia and which specific features in this population should influence decisions in pain management. MATERIALS AND METHODS: An exper...
Source: Schmerz - Category: Anesthesiology Authors: Tags: Schmerz Source Type: research
Condition:   Haemophilia Interventions:   Other: Psychology and Physiotherapy group;   Other: Placebo comparator: control group Sponsors:   Instituto de Investigacion Sanitaria La Fe;   Pfizer Recruiting
Source: - Category: Research Source Type: clinical trials
Sigilon Therapeutics is a Cambridge, MA-based biotech company developing innovative therapeutics by encapsulating cells in a novel coating that renders them invisible to the immune system. The engineered cells contained in the company’s particl...
Source: Medgadget - Category: Medical Devices Authors: Tags: Exclusive Genetics Materials Medicine Pediatrics Source Type: blogs
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