LTS-101, a one-time gene therapy designed to treat children with late infantile Batten disease, is poised to enter clinical…
Articles by Marisa Wexler, MS
Brain abnormalities in juvenile Batten disease can vary according to biological sex and age, a new study in mice…
New research of mouse models of juvenile Batten disease finds that disruptions in brain activity emerge soon after birth.
A personalized treatment called Zebronkysen led to gains in function for twin girls with juvenile Batten disease in a…
The global Batten disease community will come together Monday, June 9, to connect and spread awareness about the rare…
Blood tests to look for a specific type of abnormal immune cell called vacuolated lymphocytes may help diagnose juvenile…
Measuring how unconscious brain signals respond to different sounds may help track neurocognitive decline in people with juvenile Batten…
The enzyme replacement therapy Brineura (cerliponase alfa) is now approved by the U.S. Food and Drug Administration (FDA) as…
Note: This story was updated March 5, 2024, to clarify that Taysha Gene Therapies set aside the development of TSHA-118,…
Umbilical cord blood transplantation (UCBT) can be safely performed in children with Batten disease, a study reported. Preliminary data…