Tag: Pompe disease

Affecting millions worldwide, lysosomal storage disorders are rare genetic conditions caused by the accumulation of metabolic substrates within cells. The oral medication miglustat was developed to treat these conditions by tackling the root cause of the buildup, offering a versatile therapeutic approach.

The infusion time for Nexviazyme, an enzyme replacement therapy for Pompe disease, can vary significantly among patients, typically lasting several hours depending on dosage and individual tolerance. This intravenous treatment is administered incrementally, with initial infusions often taking longer than subsequent ones. Understanding the factors influencing **how long is a Nexviazyme infusion** is crucial for patients and their caregivers in planning their treatment schedules.

Affecting approximately 3,500 people in the US, Pompe disease is a rare genetic disorder treated by enzyme replacement therapies (ERTs). **What is Nexviazyme** and how does this second-generation treatment, developed by Sanofi, offer a new option for patients with the late-onset form of the disease?.

Affecting approximately 1 in every 57,000 people, late-onset Pompe disease is a rare genetic disorder caused by an enzyme deficiency. For many patients, the question, **'What is the drug Nexviazyme used for?'** is crucial. Nexviazyme is an enzyme replacement therapy specifically approved for treating late-onset Pompe disease in patients aged one year and older.