Although juvenile Batten disease was first described more than 200 years ago, it wasn’t until 1995 when the genetic culprit, the CLN3 gene, was identified that investigators were able to pinpoint the exact cause of the disease. By the late 1990s, rapid technological advances in the development of small animal models, light microscopy, array technology and other techniques helped investigators add significant knowledge to our understanding of the disease.
In order to translate these discoveries into therapies, BBDF has created a cohesive strategy that identifies and prioritizes knowledge gaps and action items in the pathway from here to a cure and is executing an efficient plan towards treatment for this disease. Watch the video to see how BBDF is working with research institutions, the pharmaceutical industry, and government agencies to cross the “Valley of Death” between initial scientific discoveries and meaningful progress towards a cure.
Our combined objectives are to prevent, diagnose, treat and cure juvenile Batten disease. To achieve these goals, we are engaged in multiple partnership activities with other affected families, Batten disease nonprofits, research institutes, government advisories, Pharmaceutical companies, regulatory experts and high-profile foundations committed to fighting neurodegenerative diseases