Breaking News, Trials & Filings

Polaryx Secures ODD from FDA

For PLX-200 for the treatment of Neuronal Ceroid Lipofuscinosis

Polaryx Therapeutics has received Orphan Drug Designation From the European Medicines Agency (EMA) for PLX-200 for the treatment of Neuronal Ceroid Lipofuscinosis.    Neuronal Ceroid Lipofuscinosis is a group of autosomal recessive neurodegenerative lysosomal storage disorders. This disorder is caused by cellular accumulation of abnormal auto-fluorescent lipoproteins, resulting in deterioration of neurons in both the brain and retina. Because many patients suffer from vision loss, severe...

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