Neurodegeneration with Brain Iron Accumulation (NBIA) is a group of rare genetic neurological disorders that are attracting new scientific interes due to recent advances in knowledge of the disease processes. Relevant to the NBIA disorders are the biochemistry and cell biology of mitochondrial bioenergetics, membrane homeostasis, autophagy and brain iron trafficking and regulation. Shared clinical and pathological features and intersecting pathogeneses support the continued study of the 10+ NBIA disorders as a group, with knowledge from one informing our understanding of others. Now with most of the causative genes known, this field is moving swiftly towards therapeutics. There is substantial work still needed to advance understanding of the biology of each NBIA disorder and to yield insights that will accelerate the development of rational therapeutics. A scientific conference that brings together experts in NBIA and related fields is needed in order to stimulate new research and collaborations, prioritize questions needing further investigation, and attract early career scientists to this growing field. A meeting is proposed to occur in October 2016 in the Pacific Northwest. Invited participants will include international members of the scientific community, NIH personnel, members of the family advocacy community, and industry partners. A planning committee composed of representatives will ensure that attention is paid to meeting the needs of various stakeholders and achieving the goals of this important gathering.
In the past 15 years the genetic bases for ten neurodegeneration with brain iron accumulation (NBIA) disorders have been identified, implicating common pathogeneses and revealing targets for treatment. Breakthroughs of the past few years include the discovery of novel phenotypes and causative genes as well as international efforts focused on developing rational therapeutics. We propose an international scientific conference bringing together leading scientists in the field with early career scientist interested in the NBIA disorders to help accelerate the sharing of knowledge and prioritization of work needed to improve the health of people affected by these disorders.
