See also:
Vyndamax
Vyndamax may be given to reduce death or hospitalization caused by heart problems in adults with ...
Tegsedi
Tegsedi (inotersen) is an RNA-targeting treatment that may be used to reduce the symptoms of ...
Vyndaqel
Vyndaqel (tafamidis meglumine) is a transthyretin stabilizer that may be given to reduce death or ...
Onpattro
Onpattro is used to treat polyneuropathy in adults with hereditary transthyretin-mediated ...
Wainua
Wainua (eplontersen) is used to treat polyneuropathy of hereditary transthyretin-mediated ...
Amvuttra
Amvuttra is used to treat polyneuropathy associated with hereditary transthyretin-mediated ...
Exondys 51
Exondys 51 is used to treat patients with Duchenne muscular dystrophy (DMD) who have a confirmed ...
Elevidys
Elevidys is a gene therapy for Duchenne muscular dystrophy, that is given as a one-time infusion ...
Amondys 45
Amondys 45 (casimersen) is used to treat Duchenne muscular dystrophy (DMD) with a genetic mutation ...