Enzyme Replacement Therapy

Name
Enzyme Replacement Therapy
Accession Number
DBCAT000147
Description

Not Available

Drugs
DrugDrug Description
ImigluceraseA form of recombinant human beta-glucocerebrosidase enzyme used to replace the deficient endogenous enzyme in the treatment of Gaucher disease.
AlgluceraseA form of recombinant beta-glucocerebrosidase enzyme used to replace the deficient endogenous enzyme in the treatment of Gaucher disease.
Taliglucerase alfaA hydrolytic lysosomal glucocerebroside specific enzyme, used for long term enzyme replacement therapy in patients with Type 1 Gaucher disease.
Asfotase alfaAn enzyme replacement therapy used for the treatment of perinatal/infantile and juvenile onset hypophosphatasia (HPP).
ElapegademaseA recombinant adenosine deaminase used to treat adenosine deaminase severe combined immune deficiency.
Avalglucosidase alfaA lysosomal glycogen-specific enzyme that is used to treat patients one year of age and older with late-onset Pompe disease, or lysosomal acid alpha-glucosidase (GAA) deficiency.
Agalsidase alfaA recombinant human alpha-galactosidase indicated to treat Fabry disease, a genetic deficiency in the enzyme leading to buildup of globotriaosylceramide.
Velmanase alfaA recombinant human lysosomal alpha-mannosidase used to treat non-neurological symptoms of mild to moderate alpha-mannosidosis.
Olipudase alfaA recombinant human acid sphingomyelinase used to treat Acid Sphingomyelinase Deficiency (ASMD) in children and adults.
Pegunigalsidase alfaA recombinant form of human α-galactosidase-A indicated for long-term enzyme replacement therapy in patients with Fabry disease.
Cipaglucosidase alfaA recombinant human acid alpha-glucosidase used to treat late-onset Pompe disease in adults.
Drugs & Drug Targets
DrugTargetType
ImigluceraseGlucocerebrosidetarget
AlgluceraseGlucocerebrosidetarget
Taliglucerase alfaGlucocerebrosidetarget
Asfotase alfaPyrophosphatetarget
ElapegademaseAdenosinetarget
Avalglucosidase alfaCation-independent mannose-6-phosphate receptortarget
Agalsidase alfaCation-dependent mannose-6-phosphate receptortransporter
Agalsidase alfaGlobotriaosylceramidetarget
Pegunigalsidase alfaGlobotriaosylceramidetarget
Cipaglucosidase alfaCation-independent mannose-6-phosphate receptortarget
Cipaglucosidase alfaAlpha glucosidase (GAA)target