Amondys 45(Casimersen)
Brineura(Cerliponase alfa)
Cystagon(Cysteamine)
Cystadrops(Cysteamine Ophthalmic Solution)
Evkeeza(Evinacumab)
Galafold(Migalastat)
Isturisa(Osilodrostat)
Mepsevii(Vestronidase Alfa)
Nexviazyme(Avalglucosidase Alfa)
Normosang(Human hemin)
Nulibry(Fosdenopterin)
Onpattro(Patisiran)
Palynziq(Pegvaliase-pqpz)
Strensiq(Asfotase alfa)
Skytrofa(Lonapegsomatropin-tcgd)
Spinraza(Nusinersen)
Tepezza(Teprotumumab-trbw)
Viltepso(Viltolarsen)
Voxzogo(Vosoritide)
Xermelo(Telotristat ethyl)
Uplizna(Inebilizumab-cdon)
Tavneos(Avacopan)
Symdeko(Tezacaftor/Ivacaftor & Ivacaftor)
Brinuera(Cerliponase Alfa)
