Literature DB >> 30037734

Outrageous prices of orphan drugs: a call for collaboration.

Lucio Luzzatto1, Hanna I Hyry2, Arrigo Schieppati3, Enrico Costa4, Steven Simoens5, Franz Schaefer6, Jonathan C P Roos2, Giampaolo Merlini7, Helena Kääriäinen8, Silvio Garattini9, Carla E Hollak10, Giuseppe Remuzzi11.   

Abstract

Mesh：

Year:  2018        PMID： 30037734     DOI： 10.1016/S0140-6736(18)31069-9

Source DB:  PubMed          Journal:  Lancet        ISSN： 0140-6736            Impact factor:   79.321


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  29 in total

1.  Novel Therapies for Orphan Diseases.

Authors:  José M García Fernández; Carmen Ortiz Mellet
Journal:  ACS Med Chem Lett       Date:  2019-06-18       Impact factor: 4.345

Review 2.  Clinical promise of next-generation complement therapeutics.

Authors:  Dimitrios C Mastellos; Daniel Ricklin; John D Lambris
Journal:  Nat Rev Drug Discov       Date:  2019-07-19       Impact factor: 84.694

3.  Funding and Service Organization to Achieve Universal Health Coverage for Medicines: An Economic Evaluation of the Best Investment and Service Organization for the Brazilian Scenario.

Authors:  Marina Morgado Garcia; Pamela Santos Azevedo; Andrew Mirelman; Leandro Pinheiro Safatle; Roberto Iunes; Marion Clark Bennie; Brian Godman; Augusto Afonso Guerra Junior
Journal:  Front Pharmacol       Date:  2020-04-14       Impact factor: 5.810

Review 4.  A systematic review of moral reasons on orphan drug reimbursement.

Authors:  Bettina M Zimmermann; Johanna Eichinger; Matthias R Baumgartner
Journal:  Orphanet J Rare Dis       Date:  2021-06-30       Impact factor: 4.123

Review 5.  Anti-complement Treatment for Paroxysmal Nocturnal Hemoglobinuria: Time for Proximal Complement Inhibition? A Position Paper From the SAAWP of the EBMT.

Authors:  Antonio M Risitano; Serena Marotta; Patrizia Ricci; Luana Marano; Camilla Frieri; Fabiana Cacace; Michela Sica; Austin Kulasekararaj; Rodrigo T Calado; Phillip Scheinberg; Rosario Notaro; Regis Peffault de Latour
Journal:  Front Immunol       Date:  2019-06-14       Impact factor: 7.561

6.  Relapsed/Refractory Chronic Lymphocytic Leukemia: Chemoimmunotherapy, Treatment until Progression with Mechanism-Driven Agents or Finite-Duration Therapy?

Authors:  Antonio Cuneo; Robin Foà
Journal:  Mediterr J Hematol Infect Dis       Date:  2019-03-01       Impact factor: 2.576

Review 7.  A multidisciplinary review of the policy, intellectual property rights, and international trade environment for access and affordability to essential cancer medications.

Authors:  Sangita M Baxi; Reed Beall; Joshua Yang; Tim K Mackey
Journal:  Global Health       Date:  2019-09-18       Impact factor: 4.185

8.  Efficacy and safety of D,L-3-hydroxybutyrate (D,L-3-HB) treatment in multiple acyl-CoA dehydrogenase deficiency.

Authors:  Willemijn J van Rijt; Emmalie A Jager; Derk P Allersma; A Çiğdem Aktuğlu Zeybek; Kaustuv Bhattacharya; François-Guillaume Debray; Carolyn J Ellaway; Matthias Gautschi; Michael T Geraghty; David Gil-Ortega; Austin A Larson; Francesca Moore; Eva Morava; Andrew A Morris; Kimihiko Oishi; Manuel Schiff; Sabine Scholl-Bürgi; Michel C Tchan; Jerry Vockley; Peter Witters; Saskia B Wortmann; Francjan van Spronsen; Johan L K Van Hove; Terry G J Derks
Journal:  Genet Med       Date:  2020-01-06       Impact factor: 8.822

9.  Disease awareness or subtle product placement? Orphan diseases featured in the television series "House, M.D." - a cross-sectional analysis.

Authors:  Konstantin Mechler; Juliane Rausch; William K Mountford; Markus Ries
Journal:  BMC Med Ethics       Date:  2020-03-14       Impact factor: 2.652

10.  Availability, accessibility and delivery to patients of the 28 orphan medicines approved by the European Medicine Agency for hereditary metabolic diseases in the MetabERN network.

Authors:  Jean-Michel Heard; Charlotte Vrinten; Michael Schlander; Cinzia Maria Bellettato; Corine van Lingen; Maurizio Scarpa
Journal:  Orphanet J Rare Dis       Date:  2020-01-06       Impact factor: 4.123
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