Longitudinal characterization of biomarkers for spinal muscular atrophy
- Bonati, Ulrike
- Holiga, Štefan
- Hellbach, Nicole
- Risterucci, Céline
- Bergauer, Tobias
- Tang, Wakana
- Hafner, Patricia
- Thoeni, Alain
- Bieri, Oliver
- Gerlach, Irene
- Marquet, Anne
- Khwaja, Omar
- Sambataro, Fabio
- BERTOLINO, Alessandro
- Dukart, Juergen
- Fischmann, Arne
- Fischer, Dirk
- Czech, Christian
Publication date
January 2017
DOI Publisher
Wiley ISSN
2328-9503 Language
English Citation count (estimate)
2Abstract
Recent advances in understanding Spinal Muscular Atrophy (SMA) etiopathogenesis prompted development of potent intervention strategies and raised need for sensitive outcome measures capable of assessing disease progression and response to treatment. Several biomarkers have been proposed; nevertheless, no general consensus has been reached on the most feasible ones. We observed a wide range of measures over 1 year to assess their ability to monitor the disease status and progression
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