Results 41 to 50 of about 3,868,024 (353)

First insights into the phylogenetic diversity of Mycobacterium tuberculosis in Nepal [PDF]

, 2012
BACKGROUND: Tuberculosis (TB) is a major public health problem in Nepal. Strain variation in Mycobacterium tuberculosis may influence the outcome of TB infection and disease. To date, the phylogenetic diversity of M.
A Sandgren, AE Hirsh, AN Malik, BC de Jong, Bhagwan Maharjan, Bhawana Shrestha, Bijaya Malla, C Demay, CB Holmes, CB Uwizeye, Christophe Sola, D Stucki, David Stucki, E Lasunskaia, E Svensson, ES Click, F Brossier, FA Drobniewski, G Thwaites, H Dong, H Getahun, H Han, HY Dou, I Comas, I Parwati, J Kamerbeek, JK Bonfield, JR Sun, Julia Feldmann, K Brudey, K Brudey, L Baker, L Fenner, Lukas Fenner, M Caws, M Connolly, M Coscolla, M Hanekom, Markers European Concerted Action on New Generation Genetic, MM Gutacker, MW Borgdorff, N Lari, O Neyrolles, P Supply, P Supply, R Staden, RX Liu, S Borrell, S Borrell, S Feuerriegel, S Gagneux, S Gagneux, S Narayanan, S Niemann, S Sreevatsan, Sebastien Gagneux, Sonia Borrell, T Iwamoto, T Lillebaek, TC Victor, TN Buu, TN Buu, UB Singh, US Rajapaksa, Y Hu, Y Pang, YJ Sun, YL Guo   +67 more
core   +12 more sources

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
M. Fan, A. Chan, Vincent K. C. Yan, Xinning Tong, L. K. Lau, E. Wan, Eliza Y. T. Tam, P. Ip, T. Lum, I. Wong, X. Li   +10 more
semanticscholar   +1 more source

N-acetylcysteine Provides Cytoprotection in Murine Oligodendrocytes through Heme Oxygenase-1 Activity

Biomedicines, 2020
Oligodendrocytic injury by oxidative stress can lead to demyelination, contributing to neurodegeneration. We investigated the mechanisms by which an antioxidant, N-acetylcysteine (NAC), reduces oxidative stress in murine oligodendrocytes.
Jie Zhou, Marcia R. Terluk, Lisa Basso, Usha R. Mishra, Paul J. Orchard, James C. Cloyd, Henning Schröder, Reena V. Kartha   +7 more
doaj   +1 more source

Pharmaceutical compounding of orphan active ingredients in Belgium : how community and hospital pharmacists can address the needs of patients with rare diseases [PDF]

, 2019
Background: Pharmaceutical compounding of orphan active ingredients can offer cost-effective treatment to patients when no other drug product is available for a rare disease or during periods of drug product shortages.
Boussery, Koen, De Spiegeleer, Bart, Peeters, E, Remon, Jean Paul, Van Tongelen, Inge, Vanhoorne, Valérie, Vervaet, Chris, Wynendaele, Evelien   +7 more
core   +2 more sources

A Review of Rare Disease Policies and Orphan Drug Reimbursement Systems in 12 Eurasian Countries

Frontiers in Public Health, 2020
Background: Despite international initiatives on collaboration within the field of rare diseases, patient access to orphan medicinal products (OMPs) and healthcare services differ greatly between countries.
M. Czech, Aleksandra Baran-Kooiker, K. Atikeler, Maria Demirtshyan, K. Gaitova, M. Hołownia-Voloskova, A. Turcu-Știolică, C. Kooiker, O. Piniazhko, Natella Konstandyan, Olha Zalis'ka, J. Sykut-Cegielska   +11 more
semanticscholar   +1 more source

Pressure for drug development in lysosomal storage disorders – a quantitative analysis thirty years beyond the US orphan drug act [PDF]

, 2015
Background: Lysosomal storage disorders are a heterogeneous group of approximately 50 monogenically inherited orphan conditions. A defect leads to the storage of complex molecules in the lysosome, and patients develop a complex multisystemic phenotype of
Hoffmann, Georg Friedrich, Mechler, Konstantin, Mountford, William K., Ries, Markus   +3 more
core   +1 more source

Analysis of patient access to orphan drugs in Turkey

Orphanet Journal of Rare Diseases, 2021
Background Rare diseases are life-threatening, serious, and chronic conditions that require complex care and have a low prevalence. An estimated one in 15 people worldwide are affected by rare diseases.
Güvenç Koçkaya, Sibel Atalay, Gülpembe Oğuzhan, Mustafa Kurnaz, Selin Ökçün, Çiğdem Sar Gedik, Mete Şaylan, Nazlı Şencan   +7 more
doaj   +1 more source

FDA orphan drug designations for lysosomal storage disorders – a cross-sectional analysis

PLoS ONE, 2020
Purpose To provide a quantitative clinical-regulatory insight into the status of FDA orphan drug designations for compounds intended to treat lysosomal storage disorders (LSDs). Methods Assessment of the drug pipeline through analysis of the FDA database
S. Garbade, Matthias Zielonka, K. Mechler, S. Koelker, G. Hoffmann, C. Staufner, E. Mengel, M. Ries   +7 more
semanticscholar   +1 more source

Orphan drugs revisited [PDF]

QJM, 2006
Hughes et al .1 recently discussed arguments for and against giving special funding status to orphan drugs in this journal. They concluded that there should be a uniform policy across Europe, that complete restriction was impractical, and that UK policy should aspire to the values of the EU directive.
McCabe, C., Tsuchiya, A., Claxton, K., Raftery, J.   +3 more
openaire   +4 more sources

Nuclear receptors in vascular biology [PDF]

, 2015
Nuclear receptors sense a wide range of steroids and hormones (estrogens, progesterone, androgens, glucocorticoid, and mineralocorticoid), vitamins (A and D), lipid metabolites, carbohydrates, and xenobiotics.
A Chawla, A McCurley, AA Hamers, AL Bookout, B Fisslthaler, BG Hoebel, C McCarthy, C Zhou, CH Lee, CK Glass, CK Huang, CT Lee, D Bishop-Bailey, D Bishop-Bailey, David Bishop-Bailey, DP Edwards, E Gerlach, F Akbiyik, FA Pereira, FL Wynne, FY Ali, G Pascual, I Dussault, I Spoletini, J He, J Qin, J Sonoda, J Xiang, K Spruiell, KE Swales, L Calza, L Li, L Piqueras, LA Moraes, LA Moraes, LJ Jonk, LR You, M Kaushik, M Matic, N Albermann, N Schafer, N Takamoto, Q Qin, R Zhao, S Asgary, S Chakrabarti, S Marino De, S Stein, SA Kliewer, V Zoete, W El-Sankary, WC Lau, X Wang, Y Ma, Y Sui, Y Sui, YW Hu   +56 more
core   +2 more sources