An experimental gene therapy for late infantile Batten disease showed a good safety profile and appeared to be working…
Articles by Marisa Wexler, MS
Managing fear episodes in people with juvenile Batten disease requires minimizing situations that provoke patients, and increasing their threshold…
A child with late infantile Batten disease treated with the gene therapy RGX-181 experienced a more than 80% reduction in…
Horizon Therapeutics has awarded grants to help support 50 organizations around the world that are focused on improving the…
Children with juvenile Batten disease frequently show abnormalities in eye tests, such as a bull’s-eye appearance in the macula…
A mini pig model could be an effective way to examine brain changes to track disease progression in late…
In rare cases, people with juvenile Batten disease may go years or even decades without developing seizures after the…
Mutations in genes associated with Batten disease can result in a wide range of clinical manifestations, from whole-body symptoms…
The Batten Disease Support and Research Association (BDSRA) is holding a contest to find the design for the T-shirt…
A treatment using stem cells modified to produce PPT1 — the missing enzyme in infantile Batten disease — prevented…