Natural history of Type 1 spinal muscular atrophy: a retrospective, global, multicenter study

Natural history of Type 1 spinal muscular atrophy: a retrospective, global, multicenter study

Abstract Background ANCHOVY was a global, multicenter, chart-review study that aimed to describe the natural history of Type 1 spinal muscular atrophy (SMA) from a broad geographical area and provide further contextualization of results from the FIREFISH (NCT02913482) interventional study of risdipl...

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Bibliographic Details
Main Authors: Claude Cances, Dmitry Vlodavets, Giacomo Pietro Comi, Riccardo Masson, Maria Mazurkiewicz-Bełdzińska, Kayoko Saito, Edmar Zanoteli, Angela Dodman, Muna El-Khairi, Ksenija Gorni, Isaac Gravestock, Janine Hoffart, Renata S. Scalco, Basil T. Darras, the ANCHOVY Working Group
Format: Article
Language:English
Published: BMC 2022-07-01
Series:Orphanet Journal of Rare Diseases
Subjects:
ANCHOVY
FIREFISH
SMA natural history
Type 1 SMA
Spinal muscular atrophy
Online Access:https://doi.org/10.1186/s13023-022-02455-x

Internet

https://doi.org/10.1186/s13023-022-02455-x

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