SMA-EUROPE workshop report: opportunities and challenges in developing clinical trials for spinal muscular atrophy in Europe-Reference-Cited by-同舟云学术

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SMA-EUROPE workshop report: opportunities and challenges in developing clinical trials for spinal muscular atrophy in Europe

Published:2013 Issue:1 Volume:8 Page:44
ISSN:1750-1172
Container-title:Orphanet Journal of Rare Diseases
language:en
Short-container-title:Orphanet Journal of Rare Diseases

Author:

Kayadjanian Nathalie,Burghes Arthur,Finkel Richard S,Mercuri Eugenio,Rouault Francoise,Schwersenz Inge,Talbot Kevin

Publisher

Springer Science and Business Media LLC

Subject

Pharmacology (medical),Genetics (clinical),General Medicine

Reference8 articles.

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

2. Spinal muscular atrophy

3. Childhood spinal muscular atrophy: controversies and challenges

4. New insights on the evolution of the SMN1 and SMN2 region: simulation and meta-analysis for allele and haplotype frequency calculations

5. Rescue of the spinal muscular atrophy phenotype in a mouse model by early postnatal delivery of SMN

Cited by 17 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. Nusinersen for Spinal Muscular Atrophy Type I with Chronic Respiratory Failure: A Retrospective Study in South Korea;Yonsei Medical Journal;2023

2. Nusinersen in type 1 spinal muscular atrophy: Twelve‐month real‐world data;Annals of Neurology;2019-07-08

3. Effect of new modalities of treatment on physicians’ management plan for patients with spinal muscular atrophy;Neurosciences;2019-01

4. An overview of the Cure SMA membership database: Highlights of key demographic and clinical characteristics of SMA members;Journal of Neuromuscular Diseases;2018-05-29

5. Mitochondrial disease patient motivations and barriers to participate in clinical trials;PLOS ONE;2018-05-17

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