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    Home > Biochemistry News > Biotechnology News > Improved brain 'get rid of trash' Innovative therapy approved by FDA breakthrough therapy

    Improved brain 'get rid of trash' Innovative therapy approved by FDA breakthrough therapy

    • Last Update: 2020-06-01
    • Source: Internet
    • Author: User
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    NPC is a deadly rare genetic disease and one of the most somal-storage diseases in lysosomalIt is because the NPC gene mutation causes a large amount of lipids to accumulate in tissues and organs, which cannot be digested by the lysosomes, which in turn leads to cellular stress response and toxic productionCurrently, no treatment for the disease is approved in the United States, and there is only one approved drug in EuropeOrphazyme's proprietary heat shock protein (HSP) platform is designed to create a natural system that allows proteins to work properlyIt can refold the misfolded protein into the correct conformation and restore its function, or use the lysosome to recover the protein that will not return to normal function, so that it no longer forms a toxic accumulationIt can also protect cells by inhibiting the permeability of the lysosome membrane, stabilizing the lysosome, and enabling the function of the cell to remove wasteHSP70 is a heat shock protein that has been shown to prevent protein build-up and is an essential cofactor in the process of metabolism of lipidsHSP platform restores abnormal proteins to their original conformation and function (Photo: Orphazyme official website)Arimoclomol is an oral small molecule heat shock stress-resusor developed by Orphazyme that penetrates the blood-brain barrierIt plays a role in stress cells by stimulating the cell's own heat shock response, increasing the production of HSP70, which in turn reduces the build-up of lipidsPreviously, the FDA has granted arimoclomol orphan drug eligibility, rare pediatric disease identification, and fast-track eligibilityCurrently, arimoclomol is being developed as a potential therapy for treatment stoicism for four rare diseases, including amyotrophic lateral sclerosis (ALS), the distribution of obsessive-body myocarditis (sIBM), NPC, and Gosche diseaseArimoclomol can increase the production of HSP70 and reduce the build-up of lipids (Photo: Orphazyme.com)"The FDA-granted arimoclomol breakthrough therapy has proven arimoclomol's potential in treating NPC patients." We hope to bring this product to NPC patients as soon as possible," said Kim Stratton, CEO of OrphazymeWe are expected to submit an application for the regulation of arimoclomol in the first half of 2020References:smh.com.au, Orphazyme Receives, MakeTheHeal Sym For Arimoclomol in Niemann-Pick Disease Type C (NPC), Retrieved November 19, 2019, from http://
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