SMA THERAPIES II AND BIOMARKERS

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Recent research in adults has shown that skeletal muscles express cardiac isoforms of troponin T (cTnT) in several neuromuscular diseases without cardiomyopathy. We examined whether increased serum cTnT is detectable also in SMA patients and if it correlates with disease severity. The new therapy with Nusinersen has shown to improve motor function. We evaluated whether this treatment has an effect on muscle mass, measured by serum creatinine and whether it changes cTnT. Between 03/2016 and 03/2018 we collected data of 16 patients with SMA (SMA1: 6, SMA 2 or 3: 10).

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