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First gene therapy approach for MLC leukoencephalopathy designated as orphan drug
The European Medicines Agency (EMA) has designated as an orphan drug the first gene therapy approach to treat megalencephalic leukoencephalopathy with
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Biomedical research network on rare diseases
Catalan institution for research
Department of physiological sciences
European medicines agency
Institute of neurosciences
Hebron research institute
Subcortical cysts
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