Last week, Taysha Gene Therapies announced the company has entered into license and inventory purchase agreements with Abeona Therapeutics for ABO-202, an adeno-associated virus (AAV) gene therapy for CLN1 disease ... Read more

If she were still alive, my younger sister would have celebrated her 22nd birthday today. Instead, Taylor died from CLN1 disease (Batten disease) in September 2018, more than 12 years ... Read more

A case report has described a rare case of a newborn diagnosed with CLN2 disease, or late infantile Batten disease, after a genetic analysis confirmed a disease-causing mutation in the TPP1 gene. The ... Read more

Common, non-invasive imaging techniques and a walking test used to assess patients with Batten disease in the clinic helped scientists identify a new set of biomarkers in a mouse model ... Read more

The U.S. Food and Drug Administration (FDA) has granted fast track designation to Amicus Therapeutics‘ AT-GTX-502, an investigational gene therapy for Batten disease caused by mutations in the CLN3 gene, ... Read more