The Rare Disease Treatment Center at Children's Wisconsin provides coordinated, innovative, and family-centered care for children and young adults with rare diseases and complex conditions.

Through a collaborative, multidisciplinary team, we help families navigate the challenges of rare disease treatment by connecting them with specialized expertise, advanced therapies, care coordination, education, and support. Our goal is to simplify the treatment journey and help children access the care they need through one centralized program.

Children's Wisconsin is committed to expanding access to innovative therapies for rare diseases. Depending on a child's diagnosis and treatment needs, available options may include:

• Gene therapies, including in vivo gene therapies.
• Small molecule therapies, including antisense oligonucleotide therapies.
• Enzyme replacement therapies.
• Biologic therapies.
• Other emerging treatments and clinical advances for rare diseases.

Each child receives individualized evaluation, monitoring, and follow-up care to support safe and effective treatment.

Rare diseases often affect multiple body systems and may require care from many specialists. Our program helps ease that burden by bringing experts together to provide coordinated care and ongoing support.

The Rare Disease Treatment Center offers:

• A dedicated, multidisciplinary team experienced in rare disease treatment.
• Coordinated care across specialties.
• Access to advanced therapies and emerging treatment options.
• Support navigating treatment approvals and insurance requirements.
• Education and resources for patients and families.
• Ongoing monitoring and follow-up care.

The Rare Disease Treatment Center provides a unique, coordinated approach to care for children and young adults with rare conditions.

• Collaborative Expertise: Specialists from multiple disciplines work together to develop personalized treatment plans.
• Access to Innovation: Families may have access to advanced therapies and emerging treatment options.
• Coordinated Care: A centralized program helps simplify communication, scheduling, and treatment planning.
• Family-Centered Support: We partner with families throughout the treatment journey, offering education, resources, and guidance every step of the way.

Our goal is to help children receive the right care at the right time while supporting families throughout their rare disease journey.