Amicus Therapeutics(美国)

Amicus Therapeutics

美国AmicusTherapeutics www.amicustherapeutics.comAmicus治疗是口服的，称为小分子药物的药理伴侣， 在级的方法来治疗范围广泛，包括溶酶体贮积症的疾病和??神经退行性疾病。AmicusTherapeuticsisaclinical-stagebiopharmaceuticalcompanyfocusedonthediscovery,developme nt,andcommercializationofanewclassofsmallmolecule,orally-administereddrugstotreatarangeofhumangeneticdiseases.Amicus’innovativetherapiesapplytoconditionsinwhichcrucialproteinsaredefectiveasaresultofimproperfolding.Insteadoftryingtoreplacethesecomplexproteins,Amicus’approachusespharmacologicalchaperonestoselectivelybindtothemisfoldedtargetprotein,increasestability,andhelptheproteinfoldintoitsproperthree-dimensionalshape.Thisallowstheproteintobetraffickedtothespecificlocationinthecell,whereitperformsitsintendedbiologicalfunction.Pharmacologicalchaperonetechnologyrepresentsanovel,next-generationapproachtothe