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Updated: Oct 20, 2026

Vascular Occlusion Training for Inclusion Body Myositis: A Novel Therapeutic Approach
Published on: June 5, 2010
Myosin inhibitor therapy in Fabry cardiomyopathy with dynamic left ventricular outflow obstruction
Kolja Lau1,2, Nils Petri1,3, Peter Nordbeck1,2
1Department of Internal Medicine I, University Hospital Würzburg, Würzburg, Germany.
Abstract:
A 56-year-old woman with presumed hypertrophic cardiomyopathy was diagnosed with Fabry disease (GLA: p.Glu358del) and a concomitant PLN variant. Imaging revealed advanced Fabry cardiomyopathy with dynamic LVOT obstruction. Despite optimized medical and enzyme replacement therapy, symptoms progressed (NYHA-class III) with a marked increase in LVOT gradient. Mavacamten was initiated and uptitrated under echocardiographic monitoring. At three months, LVOT gradient significantly decreased and functional status improved (NYHA-class I), while systolic function remained preserved. This case highlights the importance of detailed phenotyping and suggests that myosin inhibition may represent a therapeutic option for LVOT obstruction beyond classical sarcomeric hypertrophic cardiomyopathy.
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