Results 21 to 30 of about 32,298 (248)

Orphan drugs

Medicinski pregled, 2013
Introduction. Drugs used for treatment of rare diseases are known worldwide under the term of orphan drugs because pharmaceutical companies have not been interested in ?adopting? them, that is in investing in research, developing and producing these drugs.
Svetlana Golocorbin-Kon, Aleksandra Vojinovic, Mladena Lalic-Popovic, Nebojsa Pavlovic, Momir Mikov   +4 more
openaire   +3 more sources

Cross-national comparative study of orphan drug policies in Saudi Arabia, the United States, and the European Union

Saudi Pharmaceutical Journal, 2023
Background: Rare diseases are chronic, serious, and life-threatening conditions that have not received sufficient attention from drug developers due to their rarity.
Bander Balkhi, Asma Almuaither, Saeed Alqahtani   +2 more
doaj   +1 more source

Long way to go: Progress of orphan drug accessibility in China from 2017 to 2022

Frontiers in Pharmacology, 2023
Introduction: Over 400 million patients worldwide suffer from rare diseases. Access to orphan drugs is, therefore, crucial for this population. China has been actively working on improving orphan drug accessibility in the past decades, especially since ...
Jia Liu, Yue Yu, Yue Yu, Mingkang Zhong, Mingkang Zhong, Chunlai Ma, Chunlai Ma, Rong Shao   +7 more
doaj   +1 more source

Orphan drug: Development trends and strategies

Journal of Pharmacy and Bioallied Sciences, 2010
The growth of pharma industries has slowed in recent years because of various reasons such as patent expiries, generic competition, drying pipelines, and increasingly stringent regulatory guidelines. Many blockbuster drugs will loose their exclusivity in
Aarti Sharma, Abraham Jacob, Manas Tandon, Dushyant Kumar   +3 more
doaj   +1 more source

Pricing and reimbursement of orphan drugs: the need for more transparency

Orphanet Journal of Rare Diseases, 2011
Pricing and reimbursement of orphan drugs are an issue of high priority for policy makers, legislators, health care professionals, industry leaders, academics and patients.
Simoens Steven
doaj   +1 more source

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

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

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

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

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