The neuronal ceroid lipofuscinoses (NCLs, Batten disease) are a group of related neurodegenerative diseases mostly affecting children. As a group, the NCLs are the most common cause of childhood onset neurodegeneration in the U.S. and worldwide. Major genetic advances over the past two decades have led to the knowledge that there are at least thirteen distinct genetic forms of NCL, including several with genetic overlap with other forms of neurological disease including Parkinsonism, frontotemporal lobar dementia, and progressive myoclonic epilepsy without lysosomal storage. This has contributed key knowledge regarding NCL disease pathogenesis, and, for several subtypes of NCL, experimental therapies have now moved into clinical trial. An ?International Conference on Neuronal Ceroid Lipofuscinosis (Batten disease)? is held approximately every two years in order to convene basic and clinical researchers, clinical practitioners, and patient advocacy representatives, to discuss recent advances and strategies for further progress towards improved diagnosis and care for this group of profoundly disabling diseases. The next of these meetings, NCL2016 (www.facebook.com/NCLBoston2016/) will be held at the Wyndham Boston Beacon Hill in Boston, MA from October 5-8, 2016. This will mark the first time in over eight years that the conference will be held in the U.S. and the first time it will be located in the Greater Boston region, which has emerged as a premiere metropolitan area for cutting edge scientific research involving academic centers, biotechnology, and pharmaceutical industries. Our goal is to create a forum for vigorous scientific exchange among these diverse and complementary stakeholders in order to facilitate more rapid advancement of scientific progress towards improved diagnosis and care for patients. The scientific organizing committee has laid out topics of priority for meeting sessions, and plenary speakers from academic and industry groups are being invited. Sessions will be co-chaired by a senior investigator matched with a junior investigator, and the junior investigators will also have opportunities for presenting their research through platform presentations (to be selected from submitted abstracts) and via poster sessions throughout the conference. With the anticipated help of the patient advocacy organizations, we also aim to create a digital record of the meeting with lay summaries, in order to make the proceedings accessible to the patient/family/support community.
This conference is specifically devoted to a fatal childhood neurological disease known as neuronal ceroid lipofuscinosis (NCL) or Batten disease. The conference will serve as a unique forum for researchers, clinicians, and representatives from non-profit organizations, which are oftentimes run by parents of children affected by this devastating disease, to come together and discuss the latest advances in research, diagnostics and clinical care, and the implications of the most recent findings for developing disease-modifying treatments.
