Systemic Therapy Development in Von Hippel-Lindau Disease: An Outsized Contribution from an Orphan Disease. [PDF]
Cancers (Basel), 2022 Narayan V, Jonasch E.
europepmc +1 more source
, 1977 Series VIII: Audiocassettes features informal oral histories about the Hebrew Orphan Asylum and its alumni.Digital recordingDigital finding ...
Hebrew Orphan Asylum of New York
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Funding rare disease therapies in Australia: ensuring equitable access to health care for all Australians [PDF]
, 2014 This report argues that Australia’s system for funding new therapies that treat rare diseases is in need of reform. Overview In the last 4 years only two new therapies have been approved under Australia’s current program for rare disease therapies ...
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Orphan Does Not Always Mean Alone—A Case Study of Eosinophilic Esophagitis, an Orphan Disease
, 2023 Eosinophilic esophagitis (EoE) is a rare immunoinflammatory disease that has recently attracted significant drug development activity. Two EoE drugs have been approved, and clinical trials with several products are ongoing.
Rahnasto, Johanna Raakel
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Proteo‐transcriptomic insights into the venom composition of the ectoparasitoid Eupelmus urozonus
Pest Management Science, EarlyView.Integrated venom‐gland transcriptomics and venom proteomics of Eupelmus urozonus identified 316 venom proteins, including 75 secreted candidates, revealing diverse hydrolases, proteases/inhibitors and many uncharacterized effectors. Abstract BACKGROUND Ectoparasitoid venoms are ‘cocktails’ of bioactive molecules injected during oviposition to paralyze ...
Federica De Stefano +7 more
wiley +1 more source
Pricing Zolgensma – the world’s most expensive drug
Journal of Market Access & Health Policy, 2022 Mark Nuijten
doaj +1 more source
Bronchiectasis: moving from an orphan disease to an unpleasant socioeconomic burden. [PDF]
ERJ Open Res, 2021 Athanazio RA.
europepmc +1 more source
HOA Interview, Group, Holiday Inn, Lauderdale by the Sea (Box 90, Folder 2)
, 1987 Series VIII: Audiocassettes features informal oral histories about the Hebrew Orphan Asylum and its alumni.Digital recordingDigital finding ...
Hebrew Orphan Asylum of New York
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Can a regulatory instrument promote research and development into neglected diseases? A Policy Delphi Survey [PDF]
, 2012 Fehr A. Can a regulatory instrument promote research and development into neglected diseases? A Policy Delphi Survey. Bielefeld: University of Bielefeld; 2012.Background Rare diseases and neglected diseases are characterized by deficits in drug research ...
Fehr, Angela
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Sustainable Development, EarlyView.ABSTRACT Government food procurement investment offers significant potential to advance health and sustainability goals; yet, integrating these dimensions into policy design has proved challenging. We mapped and analysed 38 food procurement and catering‐specific policies in Australia to identify ways to strengthen policy design: 20 governing ...
Carolina Venegas Hargous +3 more
wiley +1 more source