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