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Batten Disease Neuro-infusion Program

Rady Children’s Hospital Offers New Batten Disease Neuro-infusion Program

Rady Children’s Hospital is at the forefront of fighting Batten disease, a rare neurodegenerative condition that affects children. Launched in early 2023, our Batten Disease Neuro-infusion Program provides critical intraventricular enzyme replacement therapy, slowing disease progression and offering crucial support to families.

What is Batten Disease?

Caused by genetic mutations, Batten disease gradually disrupts the brain’s ability to remove waste products, ultimately leading to cell death and a decline in various functions. Symptoms can vary between individuals, but they often include:

  • Seizures
  • Speech and motor skill delays
  • Cognitive decline
  • Vision problems
  • Movement disorders
  • Behavioral and personality changes

Rady Children’s Approach: Innovation and Compassion

Our multidisciplinary team knows the difficulties that patients and families face when it comes to Batten Disease. Here are a few of the services that we provide to treat our pediatric patients:

Targeted therapy: Enzyme replacement therapy delivered directly to the brain through an Ommaya reservoir, administered biweekly in an ambulatory setting.
Streamlined process: High-cost pharmacy processing ensures timely access to medication, while efficient insurance coverage simplifies the process for families.
Rapid diagnosis: Rady utilizes advanced genomic sequencing to expedite diagnosis, enabling early intervention and potentially slowing disease progression. A recent case highlights this success, with a patient receiving a diagnosis and starting treatment within two months.

Early intervention is key in battling Batten disease. This program offers a beacon of hope for families facing this challenging condition. By combining cutting-edge technology with compassionate care, Rady Children’s is making a significant difference in the lives of children with Batten disease.