Results 21 to 30 of about 110,217 (215)
Frontiers in Medicine, 2021 Since the implementation of the EU Orphan Regulation in 2000, the Committee for Orphan Medicinal Products at the European Medicines Agency has been evaluating the benefits of proposed orphan medicines vs. satisfactory treatment methods.
Maria E. Sheean +24 more
doaj +1 more source
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. +3 more
openaire +3 more sources
The Lancet Regional Health. Americas, 2022 Summary: Background: During the Covid-19 pandemic, children and adolescents faced poverty, potentially dying from preventable causes, or missing out essential vaccines.
Carlos Sanchez-Piedra +3 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 +7 more
core +2 more sources
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 +7 more
doaj +1 more source
Systematic review of available evidence on 11 high-priced inpatient orphan drugs [PDF]
, 2013 __Background__: Attention for Evidence Based Medicine (EBM) is growing, but evidence for orphan drugs is argued to be limited and inferior. This study systematically reviews the available evidence on clinical effectiveness, costeffectiveness and budget ...
Hakkaart-van Roijen, L. (Leona) +3 more
core +3 more sources
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 +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 +7 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 +56 more
core +3 more sources
Orphan drugs and the NHS: Should we value rarity [PDF]
, 2005 Cost effectiveness plays an important part in current decisions about the funding of health technologies. Drugs for rare disease (orphan drugs) are often expensive to produce and, by definition, will benefit only small numbers of patients.
Claxton, K., McCabe, C., Tsuchiya, A.
core +2 more sources