Amylyx Pharmaceuticals proposed mixture of sodium phenylbutyrate and tauroursodeoxycholic acid for ALS treatment.
A single dose of the elivaldogene autotemcel will cure a deadly disease.
One injection of nirsevimab reliably protects newborns against the development of RSV-associated lower respiratory tract infections.
A single infusion of valoctocogene roxaparvovec can completely cure severe hemophilia A.
One dose of betibeglogene autotemcel will permanently eliminate the dependence on regular blood transfusions.