Adeno-Associated Virus serotype 2 therapy (familial dysautonomia)

familial hypercholesterolemia program

VRX-LDLR

LeXis gene therapy (familial hypercholesterolemia)

Familial amyloidotic polyneuropathy RNAi therapy(BIOALVO)

ABCB4 gene therapy (lipid nanoparticles, progressive familial intrahepatic cholestasis type 3)

Volanesorsen(Waylivra)

Mipomersen(Kynamro)

Colchicine(Colcrys, Gloperba, Lodoco, Mitigare)

C₂₂H₂₅NO₆ · Rx

Odevixibat(Bylvay, 蓓尔唯, Kayfanda)

C₃₇H₄₈N₄O₈S₂