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FDA Grants Rare Pediatric Disease Designation to Polaryx Therapeutics' Treatment for Niemann Pick Disease


FDA Grants Rare Pediatric Disease Designation to Polaryx Therapeutics’ Treatment for Niemann Pick Disease
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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 Lee , Drug Administration , Rare Pediatric Disease , Orphan Drug , Polaryx Therapeutic , Niemann Pick Disease Types , Pick Disease , Rare Pediatric Disease Priority Review , Eiger Biopharmaceuticals , ஹான் ஜூன் லீ , ரேர் குழந்தை நோய் , ஆர்ஃபந் மருந்து , நிஎமான்ன் தேர்ந்தெடு நோய் வகைகள் , தேர்ந்தெடு நோய் , ரேர் குழந்தை நோய் ப்ரையாரிடீ விமர்சனம் , ஈகர் உயிர் மருந்துகள் ,

Polaryx Therapeutics Receives Both Rare Pediatric Disease and Orphan Drug Designations for the Treatment of Niemann Pick Disease Types A and B With PLX-300


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PARAMUS, N.J., Dec. 18, 2020 /PRNewswire/  Polaryx Therapeutics, Inc., a biotech company developing small molecule therapeutics for lysosomal storage disorders, announced today that it has received from the U.S. Food and Drug Administration (FDA) both Rare Pediatric Disease and Orphan Drug Designations for the treatment of Niemann Pick Disease Types A and B, known as Acid Sphingomyelinase Deficiency with PLX-300.
Niemann Pick Disease (NPD) Types A and B is 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 the patients only receiving supportive care. ....

Hong Kong , Hahn Jun Lee , Alex Yang , Therapeutics Inc , Drug Administration , Polaryx Therapeutics Inc , Rare Pediatric Disease , Orphan Drug Designations , Niemann Pick Disease Types , Acid Sphingomyelinase Deficiency , Pick Disease , Orphan Drug Designation , Partners Hong Kong , Jun Lee , ஹாங் காங் , ஹான் ஜூன் லீ , அலெக்ஸ் யாங் , சிகிச்சை இன்க் , ரேர் குழந்தை நோய் , நிஎமான்ன் தேர்ந்தெடு நோய் வகைகள் , தேர்ந்தெடு நோய் , ஆர்ஃபந் மருந்து பதவி , கூட்டாளர்கள் ஹாங் காங் , ஜூன் லீ ,