The Promise of Priority Review Vouchers as a Legislative Tool to Encourage Drugs for Neglected Diseases [PDF]
, 2013 Despite the intellectual property system’s success in promoting the economic well-being of the United States, this system has not achieved all socially valuable ends.
Hamming, Lesley
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The Promising Viral Threat to Bacterial Resistance: The Uncertain Patentability of Phage Therapeutics and the Necessity of Alternative Incentives [PDF]
, 2014 Bacteriophages, or “phages,” are a category of highly adept and adaptable viruses that can infect and kill bacteria. With concerns over the burgeoning antibiotic-resistance crisis looming in recent years, scientists and policymakers have expressed a ...
Todd, Kelly
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Incentives for orphan drug research and development in the United States
Orphanet Journal of Rare Diseases, 2008 Background The Orphan Drug Act (1983) established several incentives to encourage the development of orphan drugs (ODs) to treat rare diseases and conditions.
Rodriguez-Monguio Rosa +3 more
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BMC Medical Ethics, 2020 Background Approximately 7% of the general population is affected by an orphan disease, which, in the United States, is defined as affecting fewer than 1 in 1500 people. Disease awareness is often low and time-to-diagnosis delayed. Different legislations
Konstantin Mechler +3 more
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PLoS ONE, 2021 BackgroundGlioblastoma (GBM) is the most common malignant brain tumour among adult patients and represents an almost universally fatal disease. Novel therapies for GBM are being developed under the orphan drug legislation and the knowledge on the ...
Pascal Johann, Dominic Lenz, Markus Ries
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Expert Consensus on Clobazam in the Treatment of Refractory Epilepsy (2022)
Xiehe Yixue Zazhi, 2022 After regular anti-epileptic drug treatment, the symptoms of most patients with epilepsy can be well controlled or relieved, but 30%-40% of patients with epilepsy, after long-term drug treatment, still suffer from repeated seizures and develop refractory
Multi-disciplinary Team for Rare Diseases, Peking Union Medical College Hospital National Rare Diseases Committee
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Global Functional Atlas of \u3cem\u3eEscherichia coli\u3c/em\u3e Encompassing Previously Uncharacterized Proteins [PDF]
, 2009 One-third of the 4,225 protein-coding genes of Escherichia coli K-12 remain functionally unannotated (orphans). Many map to distant clades such as Archaea, suggesting involvement in basic prokaryotic traits, whereas others appear restricted to E.
Ali, Mehrab +20 more
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Patients with Gaucher disease display systemic oxidative stress dependent on therapy status
Molecular Genetics and Metabolism Reports, 2020 Gaucher disease is an autosomal recessive metabolic disorder caused by mutations in GBA1, which encodes for the lysosomal hydrolase enzyme, β-glucocerebrosidase.
Reena V. Kartha +9 more
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Systemic and Intra-Habenular Activation of the Orphan G Protein-Coupled Receptor GPR139 Decreases Compulsive-Like Alcohol Drinking and Hyperalgesia in Alcohol-Dependent Rats. [PDF]
, 2018 GPR139 is an orphan G protein-coupled receptor (GPCR) that is expressed mainly in the brain, with the highest expression in the medial habenula. The modulation of GPR139 receptor function has been hypothesized to be beneficial in the treatment of some ...
Conlisk, Dana +5 more
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PLoS ONE, 2016 BackgroundEpilepsy is a serious chronic health condition with a high morbidity impairing the life of patients and afflicted families. Many epileptic conditions, especially those affecting children, are rare disorders generating an urgent medical need for
Jan Henje Döring +3 more
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