Researchers are studying many promising treatments for Batten disease. These include medications, stem cell transplants and gene therapy to replace the gene variant. Your child’s provider might recommend participating in clinical trials (tests on humans).

2024年7月19日 · Currently, most diagnoses of Batten disease are made by genetic testing. The organizations listed below may be able to help identify a doctor familiar with NCL to aid in diagnosis. A treatment called Brineura® (cerliponase alfa) has been approved to help slow the progression of symptoms in children (age three and older) with a form of the ...

2023年5月20日 · There is no specific treatment that can cure or reverse the symptoms of any form of Batten disease but in 2017 the Food and Drug Administration approved an enzyme replacement therapy for CLN2...

2022年6月10日 · There is no cure for Batten disease, so treatment involves addressing symptoms as they arise. Around 2 to 4 in every 100,000 children in the United States are affected by this inherited...

In 2017, cerliponase alfa (Brineura), a tripeptidyl peptidase enzyme replacement therapy, became the first globally approved treatment for CLN2 Batten disease. Here, we provide an overview of the promising therapeutic avenues for Batten disease, highlighting current FDA-approved clinical trials and prospective future treatments.

How is Batten disease treated? Unfortunately, there is currently no treatment that can reverse Batten disease. However, an enzyme replacement therapy recently became available for children with CLN2 mutations that appears to slow disease progression.

Brineura is the first FDA-approved treatment to slow loss of walking ability (ambulation) in symptomatic pediatric patients 3 years of age and older with late infantile neuronal ceroid...

Early intervention with anti-seizure medications to reduce or control seizures, physical and occupational therapy to help those with Batten disease retain functioning as long as possible, and other medications to treat anxiety, depression and other challenges are also available.

2019年2月19日 · In 2017, cerliponase alfa (Brineura), a tripeptidyl peptidase enzyme replacement therapy, became the first globally approved treatment for CLN2 Batten disease. Here, we provide an overview of...

2021年8月18日 · Existing Treatments. There is an approved enzyme replacement therapy that is an option for only one form of Batten disease, which can help restore the proper breakdown of waste materials within the lysosomes. It also requires lifelong administration, which can be required as often as every two weeks via intravenous (IV) infusion.