Arimoclomol For treatment of Type-Neiman-Pique disease: Application for a new drug has been completed.
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Last Update: 2020-07-28
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Source: Internet
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Author: User
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Biopharmaceuticals is working on the development of heat shock protein response technology recently announced that it has submitted a new drug application to the United States () for the treatment of p.iNimanpike disease ()Also known as phospholipid deposition disease as congenital glycelipid metabolic disease is characterized by a large number of whole mononucleic macrophages and nervous system sphinypholipids containing neurophospholipid foam cellsIt is a small molecule heat shock stress reaction inducer that can improve the production of heat shock proteinThe heat shock reaction is a protective system that handles cellular stress and is involved in maintaining the proper folding of proteinsIt can save misfolded proteins, remove protein aggregates, and improve lysozye functionHas been awarded fast-track qualification for treatment, breakthrough therapy title and orphan medicine titleYou can have up to a few days after you complete the rolling submission to determine whether to accept the application"Data from randomized controlled clinical trials of the treatment of type C nimanpicopy support the positive effects of the drug in slowing the progression of the disease, especially in subgroups of patients over the age of four," said Professor neurology at the Mayo Clinic Children's Center in Rochester, Minnesota
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