Risdiplam distributes and increases SMN protein in both the central nervous system and peripheral organs-Reference-Cited by-同舟云学术

Risdiplam distributes and increases SMN protein in both the central nervous system and peripheral organs

Published:2018-11-29 Issue:6 Volume:6 Page:e00447
ISSN:2052-1707
Container-title:Pharmacology Research & Perspectives
language:en
Short-container-title:Pharmacol Res Perspect

Author:

Poirier Agnès¹^ORCID,Weetall Marla²,Heinig Katja¹,Bucheli Franz¹,Schoenlein Kerstin¹,Alsenz Jochem¹,Bassett Simon¹,Ullah Mohammed¹,Senn Claudia¹,Ratni Hasane¹,Naryshkin Nikolai²,Paushkin Sergey³,Mueller Lutz¹

Affiliation:

1. Roche Pharma Research and Early Development Roche Innovation Center Basel Switzerland

2. PTC Therapeutics South Plainfield New Jersey

3. SMA Foundation New York New York

Funder

Roche

Publisher

Wiley

Subject

General Pharmacology, Toxicology and Pharmaceutics,Neurology

Link

https://onlinelibrary.wiley.com/doi/pdf/10.1002/prp2.447

Reference31 articles.

1. Spinal muscular atrophy

2. A Role for SMN Exon 7 Splicing in the Selective Vulnerability of Motor Neurons in Spinal Muscular Atrophy

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

4. A single nucleotide in the SMN gene regulates splicing and is responsible for spinal muscular atrophy

5. Spinal Muscular Atrophy: More than a Disease of Motor Neurons?

Cited by 121 articles. 订阅此论文施引文献订阅此论文施引文献，注册后可以免费订阅5篇论文的施引文献，订阅后可以查看论文全部施引文献

1. Recent Progress in Gene-Targeting Therapies for Spinal Muscular Atrophy: Promises and Challenges;Genes;2024-07-30

2. Design and evaluation of antisense sequence length for modified mouse U7 small nuclear RNA to induce efficient pre-messenger RNA splicing modulation in vitro;PLOS ONE;2024-07-09

3. Insights into spinal muscular atrophy from molecular biomarkers;Neural Regeneration Research;2024-06-26

4. Male Reproduction in Spinal Muscular Atrophy (SMA) and the Potential Impact of Oral Survival of Motor Neuron 2 (SMN2) Pre-mRNA Splicing Modifiers;Neurology and Therapy;2024-05-16

5. Long-Term Comparative Efficacy and Safety of Risdiplam and Nusinersen in Children with Type 1 Spinal Muscular Atrophy;Advances in Therapy;2024-05-05