Results 31 to 40 of about 32,298 (248)

Rare diseases and orphan drugs

Annali dell'Istituto Superiore di Sanità, 2011
According to the Regulation (EC) N. 141/2000 of the European Parliament and of the Council, rare diseases are life-threatening or chronically debilitating conditions, affecting no more than 5 in 10 000 persons in the European Community.
Domenica Taruscio, Fiorentino Capozzoli, Claudio Frank   +2 more
doaj   +1 more source

Gut microbiota–derived short‐chain fatty acids regulate group 3 innate lymphoid cells in HCC

Hepatology, EarlyView., 2022
Abstract Background and Aims Type 3 innate lymphoid cells (ILC3s) are essential for host defense against infection and tissue homeostasis. However, their role in the development of HCC has not been adequately confirmed. In this study, we investigated the immunomodulatory role of short‐chain fatty acids (SCFAs) derived from intestinal microbiota in ILC3
Chupeng Hu, Bingqing Xu, Xiaodong Wang, Wen‐Hua Wan, Jinying Lu, Deyuan Kong, Yu Jin, Wenhua You, Hua Sun, Xiaoxin Mu, Dongju Feng, Yun Chen   +11 more
wiley   +1 more source

Postmarketing safety of orphan drugs: a longitudinal analysis of the US Food and Drug Administration database between 1999 and 2018

Orphanet Journal of Rare Diseases, 2022
Background Information about the specific regulatory environment of orphan drugs is scarce and inconsistent. Uncertainties surrounding the postmarketing long-term safety of orphan drugs remain. This study aimed to evaluate the labelling changes of orphan
Min Fan, Adrienne Y. L. Chan, Vincent K. C. Yan, Xinning Tong, Lauren K. W. Lau, Eric Y. F. Wan, Eliza Y. T. Tam, Patrick Ip, Terry Y. Lum, Ian C. K. Wong, X. Li   +10 more
doaj   +1 more source

Therapiekosten von Orphan Drugs vor und nach Einführung des AMNOG

Monitor Versorgungsforschung, 2022
Durch das Wegfallen des ergänzenden Bundeszuschusses im Jahr 2023 entstand eine Debatte um mögliche Maßnahmen gegen die drohende Finanzierungslücke der GKV im Jahr 2023.
Lukas Maag MSc, Dr. med. Ariane Höer, Fabian Berkemeier MSc, Dr. rer. nat. Gerrit Müller   +3 more
doaj   +1 more source

Application of orphan drug designation to cancer treatments (2008–2017): a comprehensive and comparative analysis of the USA and EU

BMJ Open, 2019
Objective To determine differences in the characteristics of cancer drugs designated as orphan drugs by the Food and Drug Administration (FDA) and European Medicines Agency (EMA).Design and setting Identification of all cancer drugs (initial or ...
Aaron S Kesselheim, Kerstin Noëlle Vokinger   +1 more
doaj   +1 more source

Using 5 consecutive years of NICE guidance to describe the characteristics and influencing factors on the economic evaluation of orphan oncology drugs

Frontiers in Public Health, 2022
ObjectiveOrphan oncology drugs used in this article were defined by the type of disease treated by drugs, as drugs used to treat rare diseases with a prevalence of ≤ 500 per million people per year.
Duan Shengnan, Lv Zixuan, Zhou Na, Zhu Weikai, Yi Yuanyuan, Liu Jiasu, Yuan Ni   +6 more
doaj   +1 more source

Engineering Approaches to Modify Immunomodulatory Functions of Mesenchymal Stromal Cells (MSCs): Tissue Regeneration and Clinical Application

Advanced Science, EarlyView.
Mesenchymal stromal cells (MSCs) show promise for treating immune‐related disorders through immunomodulation and tissue regeneration. This review gives a brief overview of current clinical approval of MSC therapies. It also discussed how bioengineering, including genetic modification, biomaterial delivery, extracellular vesicles, and iPSC‐derived MSCs,
Sichen Yang, Kejia Li, Ning Wang, Zhen Li, Zhiyong Zhang, Rocky S. Tuan, Yangzi Jiang   +6 more
wiley   +1 more source

Introducing Borsantrazole: A Trifunctional Boron‐Based Pyrazole That Extends the Lifespan of Amyotrophic Lateral Sclerosis Mice

Advanced Science, EarlyView.
Herein we report a boron‐based pyrazole, (Borsantrazole ‐ a small molecule that selectively targets oxidative stress) that significantly increases survival, reduces weight loss, delays disease onset, and affects global protein changes in the SOD1‐G37R mouse model of ALS.
Nitesh Sanghai, Rhonda Kelley, Ying Lao, M. Immanuel Reyes Madlangsakay, Prasanta Paul, M. Alejandra Llanes‐Cuesta, Jun‐Feng Wang, René P. Zahedi, Jiming Kong, Geoffrey K. Tranmer   +9 more
wiley   +1 more source

Orphans in the Market: The History of Orphan Drug Policy [PDF]

Social History of Medicine, 2017
Summary This paper examines the history of orphan drug policy, from the emergence of ‘orphans’ in the American pharmaceutical market in the 1960s, through the debates and agitations that resulted in the passage of the US Orphan Drug Act of 1983, to attempts in the 1990s to prevent abuse of that Act and restore its original intentions ...
openaire   +3 more sources

Toward Predictable Nanomedicine: Current Forecasting Frameworks for Nanoparticle–Biology Interactions

Advanced Intelligent Discovery, EarlyView.
Predictive models successfully screen nanoparticles for toxicity and cellular uptake. Yet, complex biological dynamics and sparse, nonstandardized data limit their accuracy. The field urgently needs integrated artificial intelligence/machine learning, systems biology, and open‐access data protocols to bridge the gap between materials science and safe ...
Mariya L. Ivanova, Michael Nicholls, Nicola Russo, Gueorgui Mihaylov, Konstantin Nikolic   +4 more
wiley   +1 more source