Results 21 to 30 of about 22,997 (249)

Evidence supporting regulatory-decision making on orphan medicinal products authorisation in Europe: methodological uncertainties

Orphanet Journal of Rare Diseases, 2018
Background To assess uncertainty in regulatory decision-making for orphan medicinal products (OMP), a summary of the current basis for approval is required; a systematic grouping of medical conditions may be useful in summarizing information and issuing ...
Caridad Pontes, Juan Manuel Fontanet, Roser Vives, Aranzazu Sancho, Mònica Gómez-Valent, José Ríos, Rosa Morros, Jorge Martinalbo, Martin Posch, Armin Koch, Kit Roes, Katrien Oude Rengerink, Josep Torrent-Farnell, Ferran Torres   +13 more
doaj   +1 more source

Lamellar Keratoplasty as a Method of Treatment Patients with Neurotrophic Keratopathy II and III Stages Outcome

Oftalʹmologiâ, 2022
Neurotrophic keratopathy (NTK) is an orphan, polyetiological disease that has recently been identified more frequently. Drug-resistant stages of NTC often require urgent surgical treatment.
M. Ezugbaya, I. A. Riks, S. V. Trufanov, S. Yu. Astakhov, S. S. Papanyan, L. K. Anikina, R. Boutaba, V. V. Gryaznova   +7 more
doaj   +1 more source

Identification of Tetraazacyclic Compounds as Novel Potent Inhibitors Antagonizing RORγt Activity and Suppressing Th17 Cell Differentiation. [PDF]

PLoS ONE, 2015
CD4+ T-helper cells that produce interleukin-17 (Th17 cells) are characterized as pathological T-helper cells in autoimmune diseases. Differentiation of human and mouse Th17 cells requires a key transcription regulator, retinoic acid receptor-related ...
Qingfeng Ding, Mei Zhao, Bolan Yu, Chuan Bai, Zhaofeng Huang   +4 more
doaj   +1 more source

Limitations of drug registries to evaluate orphan medicinal products for the treatment of lysosomal storage disorders [PDF]

Orphanet Journal of Rare Diseases, 2011
Orphan drugs are often approved under exceptional circumstances, requiring submission of additional data on safety and effectiveness through registries. These registries are mainly focused on one drug only and data is frequently incomplete. Some registries also address phenotypic heterogeneity and natural history data and publications on these aspects ...
Hollak, C.E.M., Aerts, J.M.F.G., Ayme, S., Manuel, J.   +3 more
openaire   +7 more sources

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

The Challenge for Orphan Drugs Remains: Three Case Studies Demonstrating the Impact of Changes to NICE Methods and Processes and Alternative Mechanisms to Value Orphan Products

PharmacoEconomics - Open, 2022
The National Institute for Health and Care Excellence (NICE) is responsible for ensuring that patients in England and Wales can access clinically and cost-effective treatments. However, NICE's processes pose significant reimbursement challenges for treatments for rare diseases.
Dawn Lee, Grant McCarthy, Omar Saeed, Rachel Allen, Kinga Malottki, Fleur Chandler   +5 more
openaire   +3 more sources

An Enzymatic Platform for Late‐Stage (Radio)isotope Labelling of Oligonucleotides With Methyltransferases

Angewandte Chemie, EarlyView.
State‐of‐the‐art synthesis of radiolabelled oligonucleotides involves multi‐step synthesis with high costs, extended timelines and significant radioactive waste generation. Herein, a dual‐methyltransferase platform enables late‐stage, site‐specific labelling, overcoming these limitations through reduced costs, faster turnaround and minimal radioactive ...
Christopher R. B. Swanson, Ulrich Kauhl, Martin R. Edelmann, Florian P. Seebeck   +3 more
wiley   +2 more sources

Overexpression of Neuron-Derived Orphan Receptor 1 (NOR-1) Rescues Cardiomyocytes from Cell Death and Improves Viability after Doxorubicin Induced Stress

Biomedicines, 2021
Following myocardial infarction, reperfusion injury (RI) is commonly observed due to the excessive formation of, e.g., reactive oxygen species (ROS).
Per-Christian Berg, Åse Mari Larsen Hansson, Øystein Røsand, Gurdeep Marwarha, Morten Andre Høydal   +4 more
doaj   +1 more source

Deciphering the pathway-specific regulatory network for production of ten-membered enediyne Tiancimycins in Streptomyces sp. CB03234-S

Microbial Cell Factories, 2022
Background The anthraquinone-fused 10-membered enediynes (AFEs), represented by tiancimycins (TNMs), possess a unique structural feature and promising potentials as payloads of antitumor antibody–drug conjugates.
Manxiang Zhu, Fan Zhang, Ting Gan, Jing Lin, Yanwen Duan, Xiangcheng Zhu   +5 more
doaj   +1 more source

Data‐Driven Materials Science for Energy‐Sustainable Applications

Advanced Materials, EarlyView.
Data‐driven approaches powered by artificial intelligence are transforming materials discovery for energy sustainability. This review examines how auto‐generated high‐quality materials databases and domain‐specific language models accelerate research in photovoltaics, thermoelectrics, batteries and magnetic materials. Applications involve extraction of
Jacqueline M. Cole
wiley   +1 more source