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12:47 EST 25th February 2020 | BioPortfolio

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Showing "Approaches Reduce Cost Impact Gene Therapies" PubMed Articles 1–25 of 37,000+

Gene Therapy for Cancer - A New Dimension and Challenge for Insurers.

:  Due to an increasingly better understanding of the human genome, the number of potential molecular targets, and therefore, potential applications by gene therapies is also increasing. After almost two decades of basic research, the first gene therapeutics are now entering the market. They are among the most expensive types of treatment in medicine. Over the next 10 years, the number and volume of their applications will increase significantly. So, our healthcare systems and inherently health insurance ...

Gene Therapy.

Gene therapy medicinal products (GTMPs) are one of the most promising biopharmaceuticals, which are beginning to show encouraging results. The broad clinical research activity has been addressed mainly to cancer, primarily to those cancers that do not respond well to conventional treatment. GTMPs to treat rare disorders caused by single-gene mutations have also made important advancements toward market availability, with eye and hematopoietic system diseases as the main applications.Nucleic acid-marketed pr...

Automation in cell and gene therapy manufacturing: from past to future.

As more and more cell and gene therapies are being developed and with the increasing number of regulatory approvals being obtained, there is an emerging and pressing need for industrial translation. Process efficiency, associated cost drivers and regulatory requirements are issues that need to be addressed before industrialisation of cell and gene therapies can be established. Automation has the potential to address these issues and pave the way towards commercialisation and mass production as it has been t...

The Impact of the New Hypertension Guidelines to Low-Dose Aspirin Prophylaxis Eligibility for the Prevention of Preeclampsia: A Cost-Benefit Analysis.

 American College of Cardiology and American Heart Association (ACC/AHA) published new guidelines which lower the cut-off for hypertension. We sought to evaluate the impact of these guidelines to cost and benefit of various low-dose aspirin prophylaxis approaches.

A Review of Economic Evaluations of Erectile Dysfunction Therapies.

Erectile dysfunction (ED) is a common and costly urologic condition with increasing prevalence as men age. Cost-effectiveness of ED therapies and whether cost-effectiveness varies for different populations of men remains underexplored.

Humanized mice are precious tools for evaluation of hematopoietic gene therapies and preclinical modeling to move towards a clinical trial.

Over the last decade, incrementally improved xenograft mouse models, which support the engraftment and development of a human hemato-lymphoid system, have been developed and represent an important fundamental and preclinical research tool. Immunodeficientmicecan be transplanted with human hematopoietic stem cells (HSCs) and this process is accompanied by HSC homing to the murine bone marrow. This is followed by stem cell expansion, multilineage hematopoiesis, long-term engraftment, and functional human anti...

Cost of disease modifying therapies for multiple sclerosis: Is front-loading the answer?

There are now over a dozen disease modifying therapies (DMTs) available to treat multiple sclerosis (MS). They vary in efficacy and safety as well as in cost. The literature on the cost effectiveness of these is often confusing and contradictory. There is a lack of quality evidence enabling the comparison of different DMTs. There are scarce randomized controlled trials which look at one DMT compared with another that is not IFN or GA. There is also a lack of systematic reviews comparing the efficacy and saf...

Impact of the Manchester Glaucoma Enhanced Referral Scheme on NHS costs.

Glaucoma filtering schemes such as the Manchester Glaucoma Enhanced Referral Scheme (GERS) aim to reduce the number of false positive cases referred to Hospital Eye Services. Such schemes can also have wider system benefits, as they may reduce waiting times for other patients. However, previous studies of the cost consequences and wider system benefits of glaucoma filtering schemes are inconclusive. We investigate the cost consequences of the Manchester GERS.

The use of biologic therapies for the management of pediatric asthma.

With better understanding of the role of type 2 inflammation in allergic asthma, there has been progress made in the development of new biologic therapies targeting these specific pathways. This review will consider diagnostic criteria for using biologic therapies for pediatric asthma with special emphasis on populations that are likely to benefit the most from particular therapies. With the exception of the anti-immunoglobulin E, omalizumab, very few studies have been published on the efficacy and safety o...

Targeting HIV-1 proviral transcription.

Despite the success of antiretroviral therapies, there is no cure for HIV-1 infection due to the establishment of a long-lived latent reservoir that fuels viral rebound upon treatment interruption. 'Shock-and-kill' strategies to diminish the latent reservoir have had modest impact on the reservoir leading to considerations of alternative approaches to target HIV-1 proviruses. This review explores approaches to target HIV-1 transcription as a way to block the provirus expression.

Pig farmers' willingness to pay for management strategies to reduce aggression between pigs.

When deciding whether to invest in an improvement to animal welfare, farmers must trade-off the relative costs and benefits. Despite the existence of effective solutions to many animal welfare issues, farmers' willingness to pay for them is largely unknown. This study modelled pig farmers' decisions to improve animal welfare using a discrete choice experiment focused on alleviating aggression between growing/finishing pigs at regrouping. Eighty-two UK and Irish pig farm owners and managers were asked to cho...

The clinical potential of gene editing as a tool to engineer cell-based therapeutics.

The clinical application of ex vivo gene edited cell therapies first began a decade ago with zinc finger nuclease editing of autologous CD4 T-cells. Editing aimed to disrupt expression of the human immunodeficiency virus co-receptor gene CCR5, with the goal of yielding cells resistant to viral entry, prior to re-infusion into the patient. Since then the field has substantially evolved with the arrival of the new editing technologies transcription activator-like effector nucleases (TALENs) and clustered regu...

Recent highlights and advances in cardiac gene therapy.

There has been significant progress in the field of gene therapy toward clinical application in recent years. Cardiac gene therapy has followed this trend, but at a slower pace compared to therapies targeting retinal, spinal, and blood disorders. New approaches targeting monogenic disorders are being developed at a rapid pace and studies in large animal models show promise. Meanwhile, several clinical trials are underway to prove the efficacy of gene therapy for cardiovascular diseases such as heart failure...

The impact of localized implementation: determining the cost-effectiveness of HIV prevention and care interventions across six United States cities.

Effective interventions to reduce the public health burden of HIV/AIDS can vary in their ability to deliver value at different levels of scale and in different epidemiological contexts. Our objective was to determine the cost-effectiveness of HIV treatment and prevention interventions implemented at previously documented scales of delivery in six US cities with diverse HIV microepidemics.

Generation of Antitumor T Cells For Adoptive Cell Therapy With Artificial Antigen Presenting Cells.

Adoptive cell therapy with ex vivo expanded tumor infiltrating lymphocytes or gene engineering T cells expressing chimeric antigen receptors (CAR) is a promising treatment for cancer patients. This production utilizes T-cell activation and transduction with activation beads and RetroNectin, respectively. However, the high cost of production is an obstacle for the broad clinical application of novel immunotherapeutic cell products. To facilitate production we refined our approach by using artificial antigen ...

Friedreich ataxia- pathogenesis and implications for therapies.

Friedreich ataxia is the most common of the hereditary ataxias. It is due to homozygous/compound heterozygous mutations in FXN. This gene encodes frataxin, a protein largely localized to mitochondria. In about 96% of affected individuals there is homozygosity for a GAA repeat expansion in intron 1 of the FXN gene. Studies of people with Friedreich ataxia and of animal and cell models, have provided much insight into the pathogenesis of this disorder. The expanded GAA repeat leads to transcriptional deficien...

Integrated assessment of management strategies for metal-contaminated dredged sediments - What are the best approaches for ports, marinas and waterways?

Sediments in ports, marinas and waterways around the world are often contaminated with metals arising from anthropogenic activities. Regular dredging is needed to achieve an appropriate water depth and reduce the environmental impact of pollutants. The aim of this study was to develop an integrated assessment method for comparing various management strategies for dredged sediments at six case study sites in Sweden. Short- and long-term environmental impacts were investigated for different management approac...

Exploring the Views of Desk-Based Office Workers and Their Employers' Beliefs Regarding Strategies to Reduce Occupational Sitting Time, with an Emphasis on Technology-Supported Approaches.

Employee and employer views regarding how technology-supported strategies can best meet their needs to reduce occupational sitting are not well known. This study explored target user and key stakeholder beliefs regarding strategies to reduce occupational sitting focusing on technology-supported approaches.

A Value of Information Analysis of Research on the 21-Gene Assay for Breast Cancer Management.

The 21-gene assay Oncotype DX (21-GA) shows promise as a guide in deciding when to initiate adjuvant chemotherapy in women with hormone receptor-positive early-stage breast cancer. Nevertheless, its routine use remains controversial, owing to insufficient evidence of its clinical utility and cost-effectiveness. Accordingly, we aim to quantify the value of conducting further research to reduce decision uncertainty in the use of the 21-GA.

Managed care opportunities and approaches to supporting appropriate selection of treatment for sight preservation.

When evaluating the impact of vision-destroying diseases, pharmacologic therapies represent a significant cost to patients, insurance providers, and society. Currently, up to 11 million people in the United States have some form of age-related macular degeneration (AMD), which is one of the leading causes of vision loss in older Americans. Ophthalmologists have administered more than 6 million intravitreal injections of aflibercept, bevacizumab, pegaptanib, and ranibizumab last year. Comprehensive assessmen...

Envisioning a Better U.S. Health Care System for All: Coverage and Cost of Care.

This paper is part of the American College of Physicians' policy framework to achieve a vision for a better health care system, where everyone has coverage for and access to the care they need, at a cost they and the country can afford. Currently, the United States is the only wealthy industrialized country that has not achieved universal health coverage. The nation's existing health care system is inefficient, unaffordable, unsustainable, and inaccessible to many. Part 1 of this paper discusses why the Uni...

Drugs, genes and screens: The ethics of preventing and treating spinal muscular atrophy.

Spinal muscular atrophy (SMA) is the most common genetic disease that causes infant mortality. Its treatment and prevention represent the paradigmatic example of the ethical dilemmas of 21st-century medicine. New therapies (nusinersen and AVXS-101) hold the promise of being able to treat, but not cure, the condition. Alternatively, genomic analysis could identify carriers, and carriers could be offered in vitro fertilization and preimplantation genetic diagnosis. In the future, gene editing could prevent th...

Pharmacological approaches to tackle NCLs.

Neuronal ceroid lipofuscinoses, also collectively known as Batten disease, are a group of rare monogenic disorders caused by mutations in at least 13 different genes. They are characterized by the accumulation of lysosomal storage material and progressive neurological deterioration with dementia, epilepsy, retinopathy, motor disturbances, and early death [1]. Although the identification of disease-causing genes provides an important step for understanding the molecular mechanisms underlying neuronal ceroid ...

Impact of complex care management on spending and utilization for high-need, high-cost Medicaid patients.

Complex care management programs have emerged as a promising model to better care for high-need, high-cost patients. Despite their widespread use, relatively little is known about the impact of these programs in Medicaid populations. This study evaluated the impact of a complex care management program on spending and utilization for high-need, high-cost Medicaid patients.

The impact of interventions for the primary prevention of hypertension in Sub-Saharan Africa: A systematic review and meta-analysis.

The prevalence of hypertension is highest in the African Region with 46% of adults aged 25 and above diagnosed with hypertension, while the lowest prevalence of 35% is found in the Americas. There is sparse evidence on the approaches used to prevent hypertension in Sub-Saharan Africa and the effectiveness of these approaches. It is therefore imperative that a systematic review; which synthesises all the available evidence on the approaches and their impact is conducted to inform public health policy and pra...

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