Marisa Wexler, MS,  senior science writer—

A personalized treatment called Zebronkysen led to gains in function for twin girls with juvenile Batten disease in a clinical study, surpassing the expectations of researchers who had hoped merely to slow disease progression. “We did not expect to see improvement in addition to a slowing of decline,” Yael…

The global Batten disease community will come together Monday, June 9, to connect and spread awareness about the rare genetic disorder. To mark International Batten Disease Awareness Day, the Batten Disease Support and Research Association (BDSRA) is planning a virtual 5K in hopes of raising $100,000 to help…

Blood tests to look for a specific type of abnormal immune cell called vacuolated lymphocytes may help diagnose juvenile Batten disease, a report showed. The study, “Vacuolated lymphocytes: a diagnostic biomarker for CLN3-related Batten disease,” was published in the Canadian Journal of Ophthalmology.  Juvenile Batten disease…

Measuring how unconscious brain signals respond to different sounds may help track neurocognitive decline in people with juvenile Batten disease, a new study suggests. The study, “Assessing the integrity of auditory sensory memory processing in CLN3 disease (Juvenile Neuronal Ceroid Lipofuscinosis (Batten disease)): an auditory evoked…

The enzyme replacement therapy Brineura (cerliponase alfa) is now approved by the U.S. Food and Drug Administration (FDA) as a treatment for CLN2 disease, also known as late infantile Batten disease, in children of all ages. The FDA previously approved Brineura to slow the loss of walking or crawling…

Note: This story was updated March 5, 2024, to clarify that Taysha Gene Therapies set aside the development of TSHA-118, along with other experimental gene therapies, to focus on another treatment for a different disorder. A patient with CLN1 disease, also known as infantile Batten disease, has received treatment…

Umbilical cord blood transplantation (UCBT) can be safely performed in children with Batten disease, a study reported. Preliminary data suggest that UCBT might be an effective treatment in some cases, particularly in people with juvenile Batten disease who are early in the course of their disease, the researchers…

An experimental gene therapy for late infantile Batten disease showed a good safety profile and appeared to be working as designed in a study done in non-human primates. “The advancement of this potential gene therapy could provide new hope to families with affected children,” Terence R. Flotte, MD, editor-in-chief…

Managing fear episodes in people with juvenile Batten disease requires minimizing situations that provoke patients, and increasing their threshold of pain and discomfort, a study reports. Sedative medications and therapies that activate specific areas of the nervous system may also help manage such responses, but solid evidence for them…

A child with late infantile Batten disease treated with the gene therapy RGX-181 experienced a more than 80% reduction in seizure frequency over the following six months, without any serious side effects. That’s according to data from a single-patient study that were announced by RGX-181’s developer, Regenxbio, in…