Amicus Therapeutics Us, Llc drugs

3 results
  • galafold - migalastat hydrochloride capsule

    (Migalastat Hydrochloride)
    Amicus Therapeutics Us, Llc
    Galafold treats adults with confirmed Fabry disease who have an amenable GLA gene variant, as determined by in vitro assay data. Approved under accelerated approval based on reduction of kidney substrate (KIC GL-3).
  • pombiliti atga - cipaglucosidase alfa - atga injection, powder, lyophilized, for solution

    (Cipaglucosidase Alfa-Atga)
    Amicus Therapeutics Us, Llc
    Pombiliti, used with Opfolda, treats late-onset Pompe disease (GAA deficiency) in adults weighing ≥40 kg who are not improving on current enzyme replacement therapy.