Results 31 to 40 of about 88,754 (289)

ORPHAN DRUG POLICY

Innovare Journal of Life Sciences, 2020
Pharmaceutical industry is crucial for mankind and contributes in some way in socio economic development of the society through jobs, community welfare, and supply chains. Indian pharmaceutical companies are one of the largest markets in the world, estimated 10% of global production and 2% of world market.
openaire   +3 more sources

Establishing a reasonable price for an orphan drug

Cost Effectiveness and Resource Allocation, 2020
Background This paper addresses the question of what a reasonable price for an orphan drug is. The research proposes a way to adjust an established payer/HTA body incremental cost-effectiveness threshold (CET) to take account of differences in patient ...
Mikel Berdud, Michael Drummond, Adrian Towse   +2 more
doaj   +1 more source

Microencapsulation, Cream Development, and Controlled Clinical Study of an Upcycled Polyphenolic Extract Combined with sh-Oligopeptide-1

Cosmetics
Olive mills produce pomace as a by-product of olive oil production process, which has a negative environmental impact. In this study, the dry extract of pomace (OG2), rich in polyphenols, was used for cosmetic purposes.
Teo Mayayo, Gabriella Russo, Ana Leticia Jiménez-Escobar, Noelia Pérez-González, Beatriz Clares, Adolfina Ruiz, Lidia Tomás-Cobos, Ana Valera, Almudena Gómez-Farto, Salvador Arias-Santiago, Trinidad Montero-Vilchez   +10 more
doaj   +1 more source

Impact of deep phenotyping: high diagnostic yield in a diverse pediatric population of 172 patients through clinical whole-genome sequencing at a single center

Frontiers in Genetics
Background: Pediatric patients with undiagnosed conditions, particularly those suspected of having Mendelian genetic disorders, pose a significant challenge in healthcare. This study investigates the diagnostic yield of whole-genome sequencing (WGS) in a
Ozlem Akgun-Dogan, Ozlem Akgun-Dogan, Ozlem Akgun-Dogan, Ecenur Tuc Bengur, Ecenur Tuc Bengur, Beril Ay, Gulsah Sebnem Ozkose, Gulsah Sebnem Ozkose, Emre Kar, Fuat Baris Bengur, Aybike S. Bulut, Aybike S. Bulut, Ayca Yigit, Ayca Yigit, Eylul Aydin, Eylul Aydin, Fatma Nisa Esen, Ozkan Ozdemir, Ozkan Ozdemir, Ahmet Yesilyurt, Yasemin Alanay, Yasemin Alanay, Yasemin Alanay   +22 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

Genetic heterogeneity in childhood leukemia/lymphoma: a Turkish cohort with strong predisposition

Frontiers in Genetics
BackgroundLeukemia is the most common cancer in children, and 10%–15% of patients with leukemia/lymphoma carry pathogenic germline cancer-predisposing variants.
Gizem Onder, Gizem Onder, Gizem Onder, Ozkan Ozdemir, Ozkan Ozdemir, Fulya Taylan, Fulya Taylan, Cengiz Canpolat, Koray Yalcin, Koray Yalcin, Fatih Erbey, Fatih Erbey, Banu Oflaz Sozmen, Banu Oflaz Sozmen, Fikret Asarcikli, Fikret Asarcikli, Turan Bayhan, Turan Bayhan, Yunus Murat Akcabelen, Nese Yarali, Nese Yarali, Namik Yasar Ozbek, Ikbal Ok Bozkaya, Dilek Kacar, Berk Ergun, Alper Akkus, Alper Akkus, Davut Albayrak, Elif Ince, Ugur Demirsoy, Gul Nihal Ozdemir, Omer Dogru, Seda Aras, Eylul Aydin, Eylul Aydin, Busra Unal, Ufuk Amanvermez, Ufuk Amanvermez, Ozlem Akgun Dogan, Ozlem Akgun Dogan, Sezer Akyoney, Muge Sayitoglu, Ann Nordgren, Ann Nordgren, Ann Nordgren, Ann Nordgren, Nihat Bugra Agaoglu, Nihat Bugra Agaoglu, Ugur Ozbek, Ugur Ozbek, Ozden Hatirnaz Ng, Ozden Hatirnaz Ng   +51 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

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   +3 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
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

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