Literature DB >> 25633169

Glybera's second act: the curtain rises on the high cost of therapy.

Seppo Ylä-Herttuala.   

Abstract

Mesh：

Year:  2015        PMID： 25633169      PMCID： PMC4445626          DOI： 10.1038/mt.2014.248

Source DB:  PubMed          Journal:  Mol Ther        ISSN： 1525-0016            Impact factor:   11.454


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  3 in total

1.  The business case for cell and gene therapies.

Authors:  Mohamed Abou-El-Enein; Gerhard Bauer; Petra Reinke
Journal:  Nat Biotechnol       Date:  2014-12       Impact factor: 54.908

2.  Endgame: glybera finally recommended for approval as the first gene therapy drug in the European union.

Authors:  Seppo Ylä-Herttuala
Journal:  Mol Ther       Date:  2012-10       Impact factor: 11.454

3.  The special case of gene therapy pricing.

Authors:  Troyen A Brennan; James M Wilson
Journal:  Nat Biotechnol       Date:  2014-09       Impact factor: 54.908
  3 in total
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1.  The payers' perspective on gene therapies.

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Journal:  Nat Biotechnol       Date:  2015-09       Impact factor: 54.908

Review 2.  Ethical development of stem-cell-based interventions.

Authors:  Amanda MacPherson; Jonathan Kimmelman
Journal:  Nat Med       Date:  2019-07-03       Impact factor: 53.440

3.  Acceptance and Access to Gene Editing: Science and Our Obligations to Mankind.

Authors:  Yuman Fong; Perry B Hackett
Journal:  Mol Ther       Date:  2016-12-28       Impact factor: 11.454

4.  Equitable Access to Gene Therapy: A Call to Action for the American Society of Gene and Cell Therapy.

Authors:  Kenneth Cornetta; Kirtika Patel; Christopher Mwaniki Wanjiku; Naftali Busakhala
Journal:  Mol Ther       Date:  2018-11-16       Impact factor: 11.454

5.  Copackaged AAV9 Vectors Promote Simultaneous Immune Tolerance and Phenotypic Correction of Pompe Disease.

Authors:  Phillip A Doerfler; Adrian G Todd; Nathalie Clément; Darin J Falk; Sushrusha Nayak; Roland W Herzog; Barry J Byrne
Journal:  Hum Gene Ther       Date:  2016-01       Impact factor: 5.695

6.  Impact of orphan drugs on Latvian budget.

Authors:  Konstantins Logviss; Dainis Krievins; Santa Purvina
Journal:  Orphanet J Rare Dis       Date:  2016-05-11       Impact factor: 4.123

7.  Profitability and Market Value of Orphan Drug Companies: A Retrospective, Propensity-Matched Case-Control Study.

Authors:  Dyfrig A Hughes; Jannine Poletti-Hughes
Journal:  PLoS One       Date:  2016-10-21       Impact factor: 3.240

Review 8.  Animal models of β-hemoglobinopathies: utility and limitations.

Authors:  Bradley McColl; Jim Vadolas
Journal:  J Blood Med       Date:  2016-11-04

Review 9.  Cell-based therapy technology classifications and translational challenges.

Authors:  Natalie M Mount; Stephen J Ward; Panos Kefalas; Johan Hyllner
Journal:  Philos Trans R Soc Lond B Biol Sci       Date:  2015-10-19       Impact factor: 6.237

Review 10.  Volanesorsen in the Treatment of Familial Chylomicronemia Syndrome or Hypertriglyceridaemia: Design, Development and Place in Therapy.

Authors:  Oluwayemisi Esan; Anthony S Wierzbicki
Journal:  Drug Des Devel Ther       Date:  2020-07-06       Impact factor: 4.162
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