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FDA Grants Rare Pediatric Disease Designation to Polaryx Therapeutics’ Treatment for Niemann Pick Disease Share Rare Daily Staff The U.S. Food and Drug Administration granted Rare Pediatric Disease and Orphan Drug designations to Polaryx Therapeutics’ PLX-300 for the treatment of Niemann Pick Disease Types A and B. Niemann Pick Disease (NPD) Types A and B are a group of ultra-rare and fatal pediatric neurodegenerative disorders caused by defects in acid sphingomyelinase, a lysosomal enzyme which plays a pivotal role in the degradation of sphingomyelin. These genetic defects result in increased intracellular accumulation of sphingomyelin leading to severe neurodegeneration, cerebellar ataxia, and dementia. There is no cure for these diseases with patients receiving only supportive care.

Hahn jun leeDrug administrationRare pediatric diseaseOrphan drugPolaryx therapeuticNiemann pick disease typesPick diseaseRare pediatric disease priority reviewEiger biopharmaceuticalsஹான் ஜூன் லீரேர் குழந்தை நோய்ஆர்ஃபந் மருந்துநிஎமான்ன் தேர்ந்தெடு நோய் வகைகள்தேர்ந்தெடு நோய்ரேர் குழந்தை நோய் ப்ரையாரிடீ விமர்சனம்ஈகர் உயிர் மருந்துகள்