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Polaryx Therapeutics Receives Both Rare Pediatric Disease and Orphan Drug Designations for the Treatment of Krabbe Disease With PLX-300

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PARAMUS, N.J., Feb. 10, 2021 /PRNewswire/ Polaryx Therapeutics, Inc., a biotech company developing small molecule therapeutics for lysosomal storage disorders, announced today that it has received both Rare Pediatric Disease and Orphan Drug Designations for the treatment of Krabbe Disease (a.k.a. Globoid Cell Leukodystrophy) with PLX-300 from the U.S. Food and Drug Administration (FDA).
Krabbe disease (KD) is a rare, heritable metabolic genetic disorder that affects approximately one in 100,000 individuals in the United States. KD is caused by deficiency of the lysosomal enzyme, galactocerebrosidase (GALC). The function of GALC is to catabolize the cytotoxic lipid, galactosylsphingosine (also known as psychosine). When either the level of GALC or its activity is compromised, galactosylsphingosine accumulates in the nervous system, ultimately leading to neurodegeneration and demyelination in patients. KD is a devastating neu ....

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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 ,