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“I have SMA, SMA doesn’t have me”: a qualitative snapshot into the challenges, successes, and quality of life of adolescents and young adults with SMA

BACKGROUND: With the approval of three treatments for spinal muscular atrophy (SMA) and several promising therapies on the horizon, the SMA adolescent and young adult populations are expected to evolve in the coming years. It is imperative to understand this cohort as it exists today to provide opti...

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Détails bibliographiques
Publié dans:Orphanet J Rare Dis
Auteurs principaux: Mazzella, Allison, Curry, Mary, Belter, Lisa, Cruz, Rosángel, Jarecki, Jill
Format: Artigo
Langue:Inglês
Publié: BioMed Central 2021
Sujets:
Accès en ligne:https://ncbi.nlm.nih.gov/pmc/articles/PMC7898731/
https://ncbi.nlm.nih.gov/pubmed/33618755
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1186/s13023-021-01701-y
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