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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Feasibility of following up gamma-hydroxybutyric acid concentrations in sodium oxybate (Xyrem®)-treated narcoleptic patients using dried blood spot sampling at home : an exploratory study [PDF]
, 2013 Background: Gamma-hydroxybutyric acid (GHB), well known as a party drug, especially in Europe, is also legally used (sodium oxybate, Xyrem (R)) to treat a rare sleep disorder, narcolepsy with cataplexy.
Hertegonne, Katrien +3 more
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ZNRD2 Mediated Nucleoprotein Aggregation Impairs Respiratory Syncytial Virus Replication
Advanced Science, EarlyView.During RSV infection, nucleoprotein (N) forms RNA‐bound oligomers. The host protein ZNRD2 binds to these oligomers, promoting their transition into insoluble aggregates. These aggregates simultaneously sequester functional N to restrict viral production and disrupt chaperonin assembly quality control by interfering with ZNRD2's role as an adaptor ...
Haiwu Zhou +8 more
wiley +1 more source
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
doaj +1 more source
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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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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XIAP Stabilizes DDRGK1 to Promote ER‐Phagy and Protects Against Noise‐Induced Hearing Loss
Advanced Science, EarlyView.Mechanism of GAS‐mediated protection against noise‐induced hearing loss (NIHL). Noise exposure activates the ATF4/eIF2α axis, downregulating XIAP and promoting DDRGK1 degradation, thereby inhibiting ER‐phagy and leading to hair cell (HC) death. GAS treatment rescues XIAP and DDRGK1 expression, reactivating ER‐phagy to mitigate HC loss, synaptic damage,
Lin Yan +13 more
wiley +1 more source
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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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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Deletion of Gpr27 in vivo reduces insulin mRNA but does not result in diabetes. [PDF]
, 2020 Gpr27 is a highly conserved, orphan G protein coupled receptor (GPCR) previously implicated in pancreatic beta cell insulin transcription and glucose-stimulated insulin secretion in vitro. Here, we characterize a whole-body mouse knockout of Gpr27. Gpr27
Chopra, Deeksha G +4 more
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