Children's Wisconsin has been designated a Qualified Treatment Center (QTC) for FAYUVI™ (rebisufligene etisparvovec-hopf), a newly approved gene therapy for Sanfilippo syndrome type A, also known as Mucopolysaccharidosis type IIIA or MPS IIIA.

The designation recognizes the expertise of the Children's Wisconsin Rare Disease Treatment Center, which brings together specialists and coordinated support to provide access to advanced therapies for children with rare genetic conditions and complex medical needs.

The U.S. Food and Drug Administration recently approved the gene therapy for eligible patients with MPS IIIA, a rare genetic disease that causes progressive damage to multiple organs including the brain and nervous system over time. With this designation, Children's Wisconsin is now part of a nationwide network of treatment centers qualified to provide this therapy and is the first Qualified Treatment Center in Wisconsin to offer it.

"For families facing a diagnosis of Sanfilippo syndrome type A, there have been no disease-modifying treatment options until now," said Michael Finkel, DO, Medical Director of the Rare Disease Treatment Center at Children's Wisconsin. "This new therapy offers something many families have been waiting for: a treatment option that targets the underlying disease and brings new hope for the future. We are proud to bring this therapy to Children’s Wisconsin and give families access to both this treatment and a team experienced in caring for children with rare and complex genetic conditions."

Sanfilippo syndrome type A affects the body's ability to break down certain sugar molecules. Over time, these substances build up in the body, especially in the brain, and cause damage. Children with the condition may develop as expected at first but later have delays in learning and development, followed by loss of speech, cognitive abilities and other skills. The neurodegenerative rare disease is ultimately fatal, and treatment has been limited to supportive care focused on symptoms, without options to address the cause of the disease.

FAYUVI™, developed by Ultragenyx, is a one-time gene therapy given through an IV infusion. It delivers a healthy copy of the gene that helps the body make an enzyme that is missing or not working properly in children with MPS IIIA. The goal of the therapy is to help the body break down harmful substances and slow the progression of the disease.

This is the second gene therapy for which Children's Wisconsin has been designated a Qualified Treatment Center. Earlier this year, Children's Wisconsin became a QTC for GENGLYCOS™, a gene therapy for glycogen storage disease type 1A (GSD Type 1A). Together, these designations reflect the organization's growing expertise in delivering advanced therapies for children with rare diseases.

The Rare Disease Treatment Center helps children and young adults with rare diseases and complex conditions access breakthrough treatments and advanced therapies with coordinated support through a single program. By bringing together experts from across specialties, the center simplifies the treatment journey for families while providing comprehensive care before, during and after treatment.

"As new treatment options become available, we want families to know they have a place to turn," said Dr. Finkel. "Our Rare Disease Treatment Center was created to make the rare disease journey easier by bringing expertise, care coordination and innovative therapies together in one place. Whether families come from Wisconsin, elsewhere in the United States or beyond, our goal is to ensure they have access to the care and support they need every step of the way.”