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

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 risdiplam treat...

Descripción completa

Detalles Bibliográficos
Autores principales: Cances, Claude, Vlodavets, Dmitry, Comi, Giacomo Pietro, Masson, Riccardo, Mazurkiewicz-Bełdzińska, Maria, Saito, Kayoko, Zanoteli, Edmar, Dodman, Angela, El-Khairi, Muna, Gorni, Ksenija, Gravestock, Isaac, Hoffart, Janine, Scalco, Renata S., Darras, Basil T.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: BioMed Central 2022
Materias:
Research
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9336055/
https://www.ncbi.nlm.nih.gov/pubmed/35906608
http://dx.doi.org/10.1186/s13023-022-02455-x

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9336055/
https://www.ncbi.nlm.nih.gov/pubmed/35906608
http://dx.doi.org/10.1186/s13023-022-02455-x

Ejemplares similares