Literature DB >> 32051111

Health ministers condemn Novartis lottery for Zolgensma, the world's most expensive drug.

Owen Dyer1.   

Abstract

Year:  2020        PMID： 32051111     DOI： 10.1136/bmj.m580

Source DB:  PubMed          Journal:  BMJ        ISSN： 0959-8138


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  7 in total

1.  Orthopaedic Gene Therapy: Twenty-Five Years On.

Authors:  Christopher H Evans; Steve C Ghivizzani; Paul D Robbins
Journal:  JBJS Rev       Date:  2021-08-26

Review 2.  Gene therapy for bone healing: lessons learned and new approaches.

Authors:  Rodolfo E De la Vega; Aysegul Atasoy-Zeybek; Joseph A Panos; Martijn VAN Griensven; Christopher H Evans; Elizabeth R Balmayor
Journal:  Transl Res       Date:  2021-05-05       Impact factor: 10.171

Review 3.  The involvement of rare disease patient organisations in therapeutic innovation across rare paediatric neurological conditions: a narrative review.

Authors:  Christina Q Nguyen; Kristine Alba-Concepcion; Elizabeth E Palmer; Jackie L Scully; Nicole Millis; Michelle A Farrar
Journal:  Orphanet J Rare Dis       Date:  2022-04-18       Impact factor: 4.303

Review 4.  Gene Therapy in Orthopaedics: Progress and Challenges in Pre-Clinical Development and Translation.

Authors:  Rachael S Watson-Levings; Glyn D Palmer; Padraic P Levings; E Anthony Dacanay; Christopher H Evans; Steven C Ghivizzani
Journal:  Front Bioeng Biotechnol       Date:  2022-06-28

5.  Ethical Perspectives on Treatment Options with Spinal Muscular Atrophy Patients.

Authors:  Crystal J J Yeo; Zachary Simmons; Darryl C De Vivo; Basil T Darras
Journal:  Ann Neurol       Date:  2022-01-25       Impact factor: 11.274

6.  [Allocation of single-use drugs in children in global compassionate use programs].

Authors:  Clemens Miller
Journal:  Ethik Med       Date:  2022-09-30       Impact factor: 0.729

Review 7.  In Search of a Cure: The Development of Therapeutics to Alter the Progression of Spinal Muscular Atrophy.

Authors:  Kristine S Ojala; Emily J Reedich; Christine J DiDonato; Stephen D Meriney
Journal:  Brain Sci       Date:  2021-02-05
  7 in total

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