GNE myopathy

Review and support for the development of a FDA dossier in patients with GNE Myopathy
Development of a manuscript describing the GNE Myopathy Functional Activity Scale (GNEM-FAS) validation
Development of a user manual for the GNEM-FAS validation
Analysis of the GNEM-FAS validation
Support for translations of the GNEM-FAS
Post-hoc analyses to aid interpretation of change in GNE Myopathy endpoints including the primary endpoint of the strength composite score

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