Tag: Enzyme replacement therapy

In clinical studies, the most commonly reported adverse reactions for Sucraid include stomach pain, vomiting, nausea, diarrhea, and constipation. This medication is used as an enzyme replacement therapy, and knowing what are the side effects of taking Sucraid is crucial for managing congenital sucrase-isomaltase deficiency (CSID) effectively.

Congenital Sucrase-Isomaltase Deficiency (CSID) is a rare genetic disorder affecting an estimated 1 in 5,000 people of European descent [1.10.2]. For those diagnosed, a key question arises: **Is there an alternative to Sucraid?**

Affecting as many as 5 in 100 people in the native communities of Canada, Alaska, and Greenland, Congenital Sucrase-Isomaltase Deficiency (CSID) requires effective management. For those prescribed Sucraid, a key question is: **how long does it take for Sucraid to work?** Most patients report improvement in digestive symptoms within the first two weeks of starting therapy.

In the United States, Phenylketonuria (PKU) affects approximately 1 in every 10,000 to 15,000 newborns. For adults with uncontrolled PKU, a key question is: **Is PALYNZIQ an enzyme replacement therapy**? While it is a form of enzyme therapy, it's more precisely called an enzyme substitution therapy.

Gaucher disease affects an estimated 1 in 50,000 to 100,000 people in the general population [1.10.4]. For those undergoing enzyme replacement therapy, a primary question is: **how long does a Cerezyme infusion take?** This intravenous treatment typically lasts between one to two hours [1.2.1, 1.2.2].

For the average patient, the annual list price of Cerezyme (imiglucerase) is approximately $200,000, though this figure can vary significantly based on dosage, disease severity, and individual factors. This high cost is typical for specialized orphan drugs designed to treat rare genetic disorders like Gaucher disease.

Over 6,000 patients worldwide are part of the International Collaborative Gaucher Group Gaucher Registry, which has provided long-term data supporting the use of Cerezyme (imiglucerase). This therapy is specifically approved for treating certain types of Gaucher disease and targets the debilitating symptoms associated with the condition. Understanding **what are the indications for Cerezyme** is crucial for patients and healthcare providers managing this rare genetic disorder.

First approved by the FDA in 2010, the medication known as VPRIV was originally manufactured by Shire plc. This article details the complex manufacturing history of VPRIV, explaining how it transitioned from its original developer to being produced by Takeda Pharmaceuticals today.

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.