Literature DB >> 29091554

The Dilemma of Two Innovative Therapies for Spinal Muscular Atrophy.

Ans T van der Ploeg1.   

Abstract

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Year:  2017        PMID： 29091554     DOI： 10.1056/NEJMe1712106

Source DB:  PubMed          Journal:  N Engl J Med        ISSN： 0028-4793            Impact factor:   91.245


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  12 in total

Review 1.  Advances in therapy for spinal muscular atrophy: promises and challenges.

Authors:  Ewout J N Groen; Kevin Talbot; Thomas H Gillingwater
Journal:  Nat Rev Neurol       Date:  2018-02-09       Impact factor: 42.937

2.  Motor neuron biology and disease: A current perspective on infantile-onset spinal muscular atrophy.

Authors:  Narendra N Jha; Jeong-Ki Kim; Umrao R Monani
Journal:  Future Neurol       Date:  2018-07-06

3.  Cost-Effectiveness of Nusinersen and Universal Newborn Screening for Spinal Muscular Atrophy.

Authors:  Ali Jalali; Erin Rothwell; Jeffrey R Botkin; Rebecca A Anderson; Russell J Butterfield; Richard E Nelson
Journal:  J Pediatr       Date:  2020-07-11       Impact factor: 4.406

4.  Preliminary Safety and Tolerability of a Novel Subcutaneous Intrathecal Catheter System for Repeated Outpatient Dosing of Nusinersen to Children and Adults With Spinal Muscular Atrophy.

Authors:  Kevin A Strauss; Vincent J Carson; Karlla W Brigatti; Millie Young; Donna L Robinson; Christine Hendrickson; Michael D Fox; Robert M Reed; Erik G Puffenberger; William Mackenzie; Freeman Miller
Journal:  J Pediatr Orthop       Date:  2018 Nov/Dec       Impact factor: 2.324

5.  Innovation for rare diseases and bioethical concerns: A thin thread between medical progress and suffering.

Authors:  Alberto Tommasini; Andrea Magnolato; Irene Bruno
Journal:  World J Clin Pediatr       Date:  2018-08-30

6.  Treatment preference among patients with spinal muscular atrophy (SMA): a discrete choice experiment.

Authors:  Alisha Monnette; Er Chen; Dongzhe Hong; Alessandra Bazzano; Stacy Dixon; W David Arnold; Lizheng Shi
Journal:  Orphanet J Rare Dis       Date:  2021-01-20       Impact factor: 4.123

Review 7.  Chemistry, mechanism and clinical status of antisense oligonucleotides and duplex RNAs.

Authors:  Xiulong Shen; David R Corey
Journal:  Nucleic Acids Res       Date:  2018-02-28       Impact factor: 16.971

8.  2017 Year in Review and Message from the Editors to Our Reviewers.

Authors:  Stefan M Pulst; Nicholas Elwood Johnson; Massimo Pandolfo; Raymond P Roos; Jeffery M Vance
Journal:  Neurol Genet       Date:  2018-02-16

9.  Spinal muscular atrophy within Amish and Mennonite populations: Ancestral haplotypes and natural history.

Authors:  Vincent J Carson; Erik G Puffenberger; Lauren E Bowser; Karlla W Brigatti; Millie Young; Dominika Korulczyk; Ashlin S Rodrigues; KaLynn K Loeven; Kevin A Strauss
Journal:  PLoS One       Date:  2018-09-06       Impact factor: 3.240

Review 10.  New and Developing Therapies in Spinal Muscular Atrophy: From Genotype to Phenotype to Treatment and Where Do We Stand?

Authors:  Tai-Heng Chen
Journal:  Int J Mol Sci       Date:  2020-05-07       Impact factor: 5.923
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