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Altuviiio (efanesoctocog alfa) is a novel and investigational recombinant factor VIII therapy with the potential to deliver near-normal factor activity levels, extending bleed protection in a once-weekly dose for people with haemophilia A.
Cerdelga (eliglustat tartrate) is a prescription medicine used for the long-term treatment of Gaucher disease type 1 (GD1) in adults who are CYP2D6 extensive metabolizers (EMs), intermediate metabolizers (IMs), or poor metabolizers (PMs).
Nexviazyme (avalglucosidase alfa) is an enzyme replacement therapy (ERT) designed to target the mannose-6-phosphate (M6P) receptor, the key pathway for uptake and transport of ERT. It is being developed for treatment of people living with pompe disease.
Altuviiio (efanesoctocog alfa) is a novel and investigational recombinant factor VIII therapy with the potential to deliver near-normal factor activity levels for most of the week, extending bleed protection in a once-weekly dose for people with haemophilia A.
Nexviadyme (avalglucosidase alfa), is the first and only newly approved medicine for Pompe disease in Europe since 2006, when the European Commission authorized the marketing of alglucosidase alfa, branded Myozyme®.
During the extension period of COMET, patients who had initially received Nexviazyme continued their treatment, while patients who were previously treated with alglucosidase alfa switched to Nexviazyme 20 mg/kg.
Nexviazyme® (avalglucosidase alfa-ngpt), is an enzyme replacement therapy designed to target the mannose6-phosphate (M6P) receptor, ) for treatment of late-onset Pompe disease in patients one year of age and older.
Nexviazyme, (avalglucosidase alfa-ngpt) has demonstrated improvements for people living with late-onset Pompe disease. In pivotal Phase 3 trial, showed improvements in respiratory function and walking distance measures in people with LOPD and established its safety profile.
Fabrazyme (agalsidase beta) works by replacing a naturally occurring enzyme (alpha-galactosidase A) to help clear GL-3 build-up in cells, including those lining the blood vessels of the kidneys, heart and skin. It is the only FDA-approved therapy for Fabry disease.
Cuprior (trientine tetrahydrochloride) is a copper chelator which eliminates absorbed copper from the body by forming a stable complex that is then eliminated through urinary excretion. It is approved for the treatment of Wilson disease.