The Orphan
Profiling the orphan enzymes
... “ orphan enzymes ” ...on orphan enzymes by mining the current content of public ...of orphan enzyme activities has decreased from 38% to 22% in ten years, there are still more than 1,000 orphans ...
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An Update on the Therapeutic Orphan
... the Orphan Products Development (OPD) program of the FDA was authorized in ...of orphan product designa- tions have a pediatric indication, and approximately half of those with marketing approval via OPD ...
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Estimating the budget impact of orphan medicines in Europe: 2010 - 2020
... In addition, the estimates from the model are supported by other related literature in this field. Figure 3 overlays the published estimates from other European researchers on the results from the study discussed here. ...
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Ethical imperatives of timely access to orphan drugs: is possible to reconcile economic incentives and patients’ health needs?
... the orphan drug legal provi- ...for orphan designation based on the 200,000 patient popula- tion threshold - authors’ estimations using data from the US Census Bureau, ...
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Estimating the clinical cost of drug development for orphan versus non-orphan drugs
... for orphan drugs included chil- dren and also included partnerships between industry, NIH and other sources as a source of ...the orphan group had a higher proportion of phase 2 trials while the ...
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Pharmaceutical expenditure on drugs for rare diseases in Canada: a historical (2007–13) and prospective (2014–18) MIDAS sales data analysis
... of orphan drug ...the orphan drug framework in Canada, which could result in an underestimation of forecasted orphan drug spend- ...future orphan drug spending, we chose a linear trend model ...
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The correlation between HTA recommendations and reimbursement status of orphan drugs in Europe
... for orphan drugs should be ...for orphan drugs are about to be implemented; currently in Poland tjust the same reimbursement procedures as for drugs used in non-rare diseases have been applied for orphans ...
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Lipid-sensors, enigmatic-orphan and orphan nuclear receptors as therapeutic targets in breast-cancer
... and Orphan- ...and Orphan-Receptors in ...the Orphan-Receptors, NR0B1, NR0B2, NR1D1, NR2F1, NR2F2 and NR4A3 exert a similar ...the Orphan-Receptors, NR2E1, NR2E3 and ...
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Impact of orphan drugs on Latvian budget
... of orphan drugs in Latvia will follow the general European tendencies and will continue to grow in the future, both in absolute num- bers and relative to the total pharmaceutical ...of orphan drugs will ...
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Orphan drugs expenditure in the Netherlands in the period 2006–2012
... on orphan drugs ...on orphan drugs to decrease from 2016 onwards ...that orphan drug prices would decrease as a consequence of ...generic orphan drugs market did not ex- pand, and it can be ...
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Creation and Development of the Public Service Orphan Drug Human Botulism Immune Globulin
... orphan disease ( ⬃ 8% of the US population) who had been left without medicines constituted a major societal lapse and public health problem that was remedied par- tially by passage of the Orphan Drug Act ...
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Does market exclusivity hinder the development of Follow-on Orphan Medicinal Products in Europe?
... an orphan designation by a sponsor are done in the late preclinical or early clinical testing stage, which doesn’t include extensive human safety test- ...authorized orphan medicinal products have been ...
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The Evolution of Orphan Regions in Genomes of a Fungal Pathogen of Wheat
... These orphan regions contained 296 genes unique to the 3D7 genome and not previously known for this ...the orphan genes were enriched in genes encoding putative effectors and included a gene that is one of ...
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Long run performance of IPOs and the role of financial analysts: some French evidence
... the orphan and non-orphan IPOs. There are 98 IPOs classified as orphan and 172 IPOs classified as ...average orphan raised ...and orphan IPOs, and the long-term debt of the ...
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Pediatric Orphan Drug Indications: 2010–2018
... drug development and also suggest that additional reforms may be needed to address the remaining unmet need for treatment among children with rare diseases. To achieve this goal, the US Congress recently passed the ...
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ORPHAN DRUGS: NEED OF AN HOUR
... (1). Orphan drugs have been defined in USA as those drugs intended to treat either a rare disease or a more common disease where the sponsor cannot make any profit ...the orphan drug shift from its ...
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Copyright in Unpublished Works: 2039 and Orphan Works
... the orphan works exception for CHIs, provided that it is for one of the above specified ...an orphan or 2039 work for purposes above and beyond that permitted by the European exception, they may apply to ...
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A Review on Recent Orphan Drugs
... the Orphan Drug Act in India ...national orphan drug regulation should offer lucrative incentive, economic outcome and market exclusivity rights to the rare drug manufacturer to enjoy the reasonable profit ...
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SRC RR 104 pdf
... the orphan list for subsequent processing, the movement onto the orphan list is done in the same transaction that advances the ...the orphan list is persistent, the orphan demon that is ...
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Incentives for orphan drug research and development in the United States
... the orphan drugs would have been approved in the absence of the ODA incentives ...for orphan drugs before the approval of the ODA ...of orphan drugs per se and as a byproduct of research for ...
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