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

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

Abstract 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. I...

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Bibliographic Details
Main Authors: Astrid Pechmann, Kirsten König, Günther Bernert, Kristina Schachtrup, Ulrike Schara, David Schorling, Inge Schwersenz, Sabine Stein, Adrian Tassoni, Sibylle Vogt, Maggie C. Walter, Hanns Lochmüller, Janbernd Kirschner
Format: Article
Language:English
Published: BMC 2019-01-01
Series:Orphanet Journal of Rare Diseases
Subjects:
Spinal muscular atrophy
Real-life outcome data
Orphan drug
Rare disease
Registry
Online Access:http://link.springer.com/article/10.1186/s13023-019-0998-4

Internet

http://link.springer.com/article/10.1186/s13023-019-0998-4

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