Results 21 to 30 of about 22,199 (155)
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 +13 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 +4 more
doaj +1 more source
The Orphan Drug Act and the Development of Stem Cell-Based Products for Rare Diseases [PDF]
Cell Stem Cell, 2010 The Orphan Drug Act encourages the development of products for rare diseases and conditions. Many conditions that stand to benefit from stem cell-based products are rare diseases. We address the Orphan Drug Act in relation to the development of stem cell-based products.
Freeman, Scott N. +3 more
openaire +2 more sources
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. +3 more
openaire +7 more sources
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 +5 more
openaire +3 more sources
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 +4 more
doaj +1 more source
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 +5 more
doaj +1 more source
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
Regulation of Antimycin Biosynthesis Is Controlled by the ClpXP Protease
mSphere, 2020 The survival of any microbe relies on its ability to respond to environmental change. Use of extracytoplasmic function (ECF) RNA polymerase sigma (σ) factors is a major strategy enabling dynamic responses to extracellular signals.
Bohdan Bilyk +4 more
doaj +1 more source
Pharmaceutical lobbying in Brazil: a missing topic in the public health research agenda
Revista de Saúde PúblicaIn the US, where registration of lobbyists is mandatory, the pharmaceutical industry and private health-care providers spend huge amounts of money seeking to influence health policies and government decisions. In Brazil, where lobbying lacks transparency,
Francisco José Roma Paumgartten
doaj +1 more source