Real-world safety and effectiveness of nusinersen, a treatment for spinal muscular atrophy, in 401 Japanese patients: results from an interim analysis of post-marketing surveillance-Reference-Cited by-同舟云学术

Real-world safety and effectiveness of nusinersen, a treatment for spinal muscular atrophy, in 401 Japanese patients: results from an interim analysis of post-marketing surveillance

Published:2022-07-27 Issue: Volume: Page:1-10
ISSN:0020-7454
Container-title:International Journal of Neuroscience
language:en
Short-container-title:International Journal of Neuroscience

Author:

Tachibana Yosuke¹^ORCID,Takasaki Sakura¹^ORCID,Hoshino Misuzu¹^ORCID,Makioka Haruki¹^ORCID,Jin Mingshou¹^ORCID

Affiliation:

1. Biogen Japan, Tokyo, Japan

Publisher

Informa UK Limited

Subject

General Medicine,General Neuroscience

Link

https://www.tandfonline.com/doi/pdf/10.1080/00207454.2022.2095270

Reference19 articles.

1. Spinal Muscular Atrophy: A Clinical and Research Update

2. Identification and characterization of a spinal muscular atrophy-determining gene

3. The human centromeric survival motor neuron gene (SMN2) rescues embryonic lethality in Smn-/- mice and results in a mouse with spinal muscular atrophy

4. A degron created by SMN2 exon 7 skipping is a principal contributor to spinal muscular atrophy severity

5. Mildly affected patients with spinal muscular atrophy are partially protected by an increased SMN2 copy number

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1. Later efficacy of nusinersen treatment in adult patients with spinal muscular atrophy: A retrospective case study with a median 4-year follow-up;Brain and Development;2024-01

2. Nusinersen demonstrates effectiveness in treating spinal muscular atrophy: findings from a three-year nationwide study in Korea;Frontiers in Neurology;2023-12-20

3. Nusinersen induces detectable changes in compound motor action potential response in spinal muscular atrophy type 1 patients with severe impairment of motor function;Brain and Development;2023-12

4. Safety and effectiveness of nusinersen, a treatment for spinal muscular atrophy, in 524 patients: results from an interim analysis of post-marketing surveillance in Japan;International Journal of Neuroscience;2023-09-07