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.
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.
Teclistamab by Johnson & Johnson is a new hope for refractory or relapsing multiple myeloma.
Ensifentrine, an inhaled PDE3/PDE4 inhibitor, in the treatment of chronic obstructive pulmonary disease.
Tofersen and the mixed results of treatment for the deadly neurodegenerative disease.