SMArtCARE - A platform to collect real-life outcome data of patients with spinal muscular atrophy

BACKGROUND: Survival and quality of life for patients affected by spinal muscular atrophy (SMA) are thought to have improved over the last decade due to changes in care. In addition, targeted treatments for SMA have been developed based on a better understanding of the molecular pathology. In 2016 a...

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Détails bibliographiques
Publié dans:Orphanet J Rare Dis
Auteurs principaux: Pechmann, Astrid, König, Kirsten, Bernert, Günther, Schachtrup, Kristina, Schara, Ulrike, Schorling, David, Schwersenz, Inge, Stein, Sabine, Tassoni, Adrian, Vogt, Sibylle, Walter, Maggie C., Lochmüller, Hanns, Kirschner, Janbernd
Format: Artigo
Langue:Inglês
Publié: BioMed Central 2019
Sujets:
Research
Accès en ligne:https://ncbi.nlm.nih.gov/pmc/articles/PMC6341722/
https://ncbi.nlm.nih.gov/pubmed/30665421
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1186/s13023-019-0998-4
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