Literature DB >> 33177183

Efficacy and costs of spinal muscular atrophy drugs.

Jonathan J Darrow1, Monica Sharma2, Mansa Shroff3, Anita K Wagner4,5.   

Abstract

Evaluating the benefits, risks, and costs of two drugs to treat spinal muscular atrophy raises questions about the future of rare disease medicines.
Copyright © 2020 The Authors, some rights reserved; exclusive licensee American Association for the Advancement of Science. No claim to original U.S. Government Works.

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Year:  2020        PMID： 33177183     DOI： 10.1126/scitranslmed.aay9648

Source DB:  PubMed          Journal:  Sci Transl Med        ISSN： 1946-6234            Impact factor:   17.956


  5 in total

1.  Analysis of Supportive Evidence for US Food and Drug Administration Approvals of Novel Drugs in 2020.

Authors:  Mayookha Mitra-Majumdar; Simon J Gunter; Aaron S Kesselheim; Beatrice L Brown; Krysten W Joyce; Murray Ross; Catherine Pham; Jerry Avorn; Jonathan J Darrow
Journal:  JAMA Netw Open       Date:  2022-05-02

Review 2.  Revisiting the role of mitochondria in spinal muscular atrophy.

Authors:  Rachel James; Helena Chaytow; Leire M Ledahawsky; Thomas H Gillingwater
Journal:  Cell Mol Life Sci       Date:  2021-04-05       Impact factor: 9.261

3.  Targeted epigenetic repression by CRISPR/dSaCas9 suppresses pathogenic DUX4-fl expression in FSHD.

Authors:  Charis L Himeda; Takako I Jones; Peter L Jones
Journal:  Mol Ther Methods Clin Dev       Date:  2020-12-10       Impact factor: 6.698

4.  Ultraexpensive gene therapies, industry interests and the right to health: the case of onasemnogene abeparvovec in Brazil.

Authors:  Adriana Mitsue Ivama-Brummell; Anita K Wagner; Vera Lúcia Edais Pepe; Huseyin Naci
Journal:  BMJ Glob Health       Date:  2022-03

Review 5.  Spinal muscular atrophy: From approved therapies to future therapeutic targets for personalized medicine.

Authors:  Helena Chaytow; Kiterie M E Faller; Yu-Ting Huang; Thomas H Gillingwater
Journal:  Cell Rep Med       Date:  2021-07-21
  5 in total

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