Results 101 to 110 of about 3,868,024 (353)

N6‐methyladenosine‐regulated exosome biogenesis orchestrates an immunosuppressive pre‐metastatic niche in gastric cancer peritoneal metastasis

Cancer Communications, EarlyView.
Abstract Background Gastric cancer peritoneal metastasis is clinically challenging, given the limited treatment options and poor prognosis. The molecular mechanisms that precede gastric cancer peritoneal metastasis, known as the pre‐metastatic niche (PMN), and its relationship with N6‐methyladenosine (m6A) modification remain unclear.
Song Li, Jianyuan Zhou, Shuang Wang, Qian Yang, Shulun Nie, Chunwang Ji, Xue Zhang, Shuhan Li, Xuanyu Zhou, Jiahui Chu, Xuehui Wu, Jianqiao Jiao, Ruitao Xu, Qian Xu, Miao Huang, Qiushi Wang, Liliang Dou, Qinqin Hu, Fan Jiang, Xin Dai, Zhaodi Nan, Xinyu Song, Di Zhang, Lian Liu   +23 more
wiley   +1 more source

The impact of orphan drug policies in treating rare diseases.

Health Information and Libraries Journal, 2019
This paper is based on Shalini Weerasooriya's Masters dissertation in Public Health at the University of Sheffield. A literature review was conducted to understand the role that orphan drug policies have played in the development of new treatments for ...
S. Weerasooriya
semanticscholar   +1 more source

Can Standard Health Technology Assessment Approaches Help Guide the Price of Orphan Drugs in Canada? A Review of Submissions to the Canadian Agency for Drugs and Technologies in Health Common Drug Review

ClinicoEconomics and Outcomes Research, 2020
Orphan drugs have high acquisition costs and when standard health technology assessment (HTA) approaches are used to assess their cost-effectiveness, they often appear not cost-effective.
C. Balijepalli, L. Gullapalli, E. Druyts, K. Yan, K. Desai, S. Barakat, J. Locklin   +6 more
semanticscholar   +1 more source

Ubiquitination in cancer: mechanisms and therapeutic opportunities

Cancer Communications, EarlyView.
Abstract Ubiquitination, a key post‐translational modification, plays an essential role in tumor biology by regulating fundamental cellular processes, such as metabolism and cell death. Additionally, it interacts with other post‐translational modifications, which are closely linked to tumorigenesis, tumor progression, the tumor microenvironment, and ...
Susi Zhu, Xu Zhang, Waner Liu, Zhe Zhou, Siyu Xiong, Jie Li, Xiang Chen, Cong Peng   +7 more
wiley   +1 more source

The prevalence and cost of unapproved uses of top-selling orphan drugs. [PDF]

PLoS ONE, 2012
The Orphan Drug Act encourages drug development for rare conditions. However, some orphan drugs become top sellers for unclear reasons. We sought to evaluate the extent and cost of approved and unapproved uses of orphan drugs with the highest unit sales ...
Aaron S Kesselheim, Jessica A Myers, Daniel H Solomon, Wolfgang C Winkelmayer, Raisa Levin, Jerry Avorn   +5 more
doaj   +1 more source

Enzyme replacement therapies: What is the best option? [PDF]

, 2018
Despite many beneficial outcomes of the conventional enzyme replacement therapy (ERT), several limitations such as the high-cost of the treatment and various inadvertent side effects including the occurrence of an immunological response against the ...
Barar, Jaleh, Khiavi, Mostafa Akbarzadeh, Mousavi, Rahimeh, Rafi, Mohammad, Safary, Azam   +4 more
core   +2 more sources

FRAME: Framework for Real‐World Evidence Assessment to Mitigate Evidence Uncertainties for Efficacy/Effectiveness – An Evaluation of Regulatory and Health Technology Assessment Decision Making

Clinical Pharmacology &Therapeutics, EarlyView.
Real‐World Evidence (RWE) is increasingly used in submissions to regulatory agencies and health technology assessment bodies (HTAbs) to support the efficacy and effectiveness of new medicines and indications. However, there is limited information on the RWE characteristics that impact its role in approval and reimbursement decisions.
Gianmario Candore, Claire Martin, Mack J. Mills, Annabel Suter, Anna Lloyd, Danitza Chavez‐Montoya, Diego Civitelli, Birgit Wolf, Paul Bolot, Juergen Wasem, Montse Soriano Gabarró, Panos G. Kanavos, Mark Sculpher   +12 more
wiley   +1 more source

Relationship between Dose, Factor IX Activity Levels and Bleeding Probability for rIX‐FP Prophylaxis in Hemophilia B: A Repeated Time‐to‐Event Analysis

Clinical Pharmacology &Therapeutics, EarlyView.
In hemophilia B, pharmacokinetic (PK)‐guided dosing of extended half‐life factor IX (EHL‐FIX) concentrates can secure targeted FIX exposure. Target FIX activity levels in plasma should be individually set primarily taking bleeding tendency into account, alongside the presence of target joints, physical activity, and preferred dosing schedules. In other
Sjoerd F. Koopman, Marjon H. Cnossen, Ron A.A. Mathot, on behalf of the OPTI‐CLOT study group and SYMPHONY consortium, S. H. Reitsma   +4 more
wiley   +1 more source

Challenges in Orphan Drug Development: Identification of Effective Therapy for Thyroid-Associated Ophthalmopathy.

Annual Review of Pharmacology and Toxicology, 2019
Thyroid-associated ophthalmopathy (TAO), the ocular manifestation of Graves' disease, is a process in which orbital connective tissues and extraocular muscles undergo inflammation and remodeling. The condition seems to result from autoimmune responses to
Terry J. Smith
semanticscholar   +1 more source

Does Misery Love Company? Evidence from pharmaceutical markets before and after the Orphan Drug Act [PDF]

With substantial fixed costs of drug development, more common conditions can support more products. If additional pharmaceutical products are beneficial, they will attract greater consumption and promote better health, e.g. greater longevity.
Frank R. Lichtenberg, Joel Waldfogel
core