Riaan Research Initiative

Research projects - Riaan Research Initiative is dedicated to advancing, accelerating, and funding translational scientific Research to generate treatments for severe and lethal neurodegenerative genetic disorders that impact the pediatric population such as cockayne syndrome. The organization's programmatic areas include supporting the development of cockayne syndrome gene replacement therapy, and drug repurposing screens by partnering with researchers and academic institutions and issuing grants. Gene therapy work involves accelerating the development of adenovirus vectors and supporting pre-clinical to clinical Research studies; the organization's drug repurposing projects involve screening small molecules to identify drugs that can halt or reverse neurodegeneration in this patient population. The organization also provides education and resources to the medical and rare disease community, the media, as well as members of the public on advancements in scientific Research and general cockayne syndrome awareness and maintains a patient registry to gather relevant clinical data for future trials.