See also:
Vyndamax
Vyndamax may be given to reduce death or hospitalization caused by heart problems in adults with ...
Attruby
Attruby (acoramidis) tablets are used to treat cardiomyopathy of wild-type or hereditary ...
Wainua
Wainua (eplontersen) is used to treat polyneuropathy of hereditary transthyretin-mediated ...
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 ...
Zolgensma
Zolgensma (onasemnogene abeparvovec-xioi) is gene therapy used for spinal muscular atrophy. It is a ...
Evrysdi
Evrysdi is used to treat spinal muscular atrophy (SMA) in adults, children, and infants. It is ...