Literature DB >> 31381549

Zolgensma - one-time gene therapy for spinal muscular atrophy.

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Abstract

Entities:  

Keywords:  Spinraza; adverse effects; dosage; efficacy; nusinersen; onasemnogene abeparvovec; safety; spinal muscular atrophy; zolgensma

Mesh：

Substances：

Year:  2019        PMID： 31381549

Source DB:  PubMed          Journal:  Med Lett Drugs Ther        ISSN： 0025-732X            Impact factor:   1.909


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  4 in total

Review 1.  Viral Vector Systems for Gene Therapy: A Comprehensive Literature Review of Progress and Biosafety Challenges.

Authors:  Sumit Ghosh; Alex M Brown; Chris Jenkins; Katie Campbell
Journal:  Appl Biosaf       Date:  2020-03-01

Review 2.  U7 snRNA: A tool for gene therapy.

Authors:  Ankur Gadgil; Katarzyna Dorota Raczyńska
Journal:  J Gene Med       Date:  2021-02-23       Impact factor: 4.565

Review 3.  RNA-Targeted Therapies and High-Throughput Screening Methods.

Authors:  Siran Zhu; Saul Rooney; Gracjan Michlewski
Journal:  Int J Mol Sci       Date:  2020-04-23       Impact factor: 5.923

4.  Modelling the Cost-Effectiveness and Budget Impact of a Newborn Screening Program for Spinal Muscular Atrophy and Severe Combined Immunodeficiency.

Authors:  Sophy T F Shih; Elena Keller; Veronica Wiley; Michelle A Farrar; Melanie Wong; Georgina M Chambers
Journal:  Int J Neonatal Screen       Date:  2022-07-20
  4 in total

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