Biomarkers predicting motor function in SMA

Presented By
Prof. Valeria Sansone, University of Milano, Italy
Trial
Phase 2, NURTURE
Conference
EAN 2020

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Sustained benefits of avalglucosidase alfa in late-onset Pompe disease

Presented By
Dr Benedikt Schoser, Ludwig-Maximilians-University of Munich, Germany
Trial
NEO1; NEO-EXT
Conference
EAN 2020

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Efficacy and safety of rituximab in refractory MG corroborated

Presented By
Dr A Dos Santos, University Hospital of Nantes, France
Conference
EAN 2020

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First-ever effective and safe treatment of CMT1A

Presented By
Dr Florian Thomas, Hackensack University Medical Center, New York, USA
Trial
Phase 3, PLEO-CMT
Conference
AAN 2019

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