Amicus Therapeutics(美國(guó))

Amicus Therapeutics

美國(guó)AmicusTherapeutics www.amicustherapeutics.comAmicus治療是口服的，稱為小分子藥物的藥理伴侶， 在級(jí)的方法來(lái)治療范圍廣泛，包括溶酶體貯積癥的疾病和??神經(jīng)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