Results 31 to 40 of about 68,610 (290)

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

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 expanding range of targets for kynurenine metabolites of tryptophan [PDF]

, 2013
The kynurenine pathway of tryptophan metabolism accounts for most of the tryptophan that is not committed to protein synthesis and includes compounds active in the nervous and immune systems.
Darlington, L. Gail, Stone, Trevor W., Stoy, Nicholas   +2 more
core   +1 more source

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

Access to Life-Saving Medicines and Intellectual Property Rights: An Ethical Assessment [PDF]

, 2011
© 2011 Cambridge University Press. Online edition of the journal is available at http://journals.cambridge.org/action/displayJournal?jid=CQHDying before one’s time has been a prominent theme in classic literature and poetry.
Aquinas, Aquinas, Aquinas, Brown, DORIS SCHROEDER, Finnis, Jentzsch, Leisinger, Locke, Mamunes, May, Nozick, Pang, PETER SINGER, Pogge, Pogge, Rawls, Rifkin, Schroeder, Selgelid, Shelley   +20 more
core   +2 more sources

Deletion of Gpr27 in vivo reduces insulin mRNA but does not result in diabetes. [PDF]

, 2020
Gpr27 is a highly conserved, orphan G protein coupled receptor (GPCR) previously implicated in pancreatic beta cell insulin transcription and glucose-stimulated insulin secretion in vitro. Here, we characterize a whole-body mouse knockout of Gpr27. Gpr27
Chopra, Deeksha G, Hennings, Thomas G, Ku, Gregory M, Yiv, Nicholas, Zhang, Yaohuan   +4 more
core  

Endocannabinoid system and anticancer properties of cannabinoids [PDF]

, 2016
Cannabinoids impact human body by binding to cannabinoids receptors (CB1 and CB2). The two main phytocannabinoids are Δ9-tetrahydrocannabinol (THC) and cannabidiol (CBD).
Nowak, Agnieszka, Słomski, Ryszard, Zeyland, Joanna, Śledziński, Paweł   +3 more
core   +2 more sources

Orphan drugs: Unmet societal need for non-profitable privately supplied new products

Research Policy, 2007
Due to the severity of rare diseases, the societal need for biopharmaceutical treatments for these diseases is high, despite low numbers of patients. Therefore, we investigated the barriers currently hindering the willingness to develop orphan drugs in the Netherlands.
Moors, E.H.M., Faber, J.
openaire   +3 more sources

Deconvolution of complex G protein-coupled receptor signaling in live cells using dynamic mass redistribution measurements [PDF]

, 2010
Label-free biosensor technology based on dynamic mass redistribution (DMR) of cellular constituents promises to translate GPCR signaling into complex optical 'fingerprints' in real time in living cells.
Blattermann, S, Drewke, C., Gomeza, J., Hennen, S., Janssen, N., Kebig, A., Kostenis, E., Merten, N., Milligan, G., Mohr, K., Mohr-Andra, M., Muller, A., Schmidt, J., Schroder, R., Smith, N.J., Wenzel, J., Zahn, S.   +16 more
core   +1 more source