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Areas for future study—Unmet needs in GNE myopathy.
| • Lack of phenotypically appropriate animal model • Reliable antibodies to GNE protein not available • Doubts about whether hyposialylation is the causative mechanism • Lack of reliable biomarkers (MRI vs. lectin staining vs. biochemical serum markers) • Resolution of common and difficult to interpret variants of unknown significance • Need for better assays to measure GNE enzymatic function |
