Background: Children and Adolescents with rare neurogenetic disorders often have no known cure or disease modifying treatments. Recent advancements in treatments are offering much needed hope to these patients and families. However, these treatments are extremely costly, have complex administration requirements and have many unknown long-term risks and outcomes. Methods: In this presentation, we will discuss our experiences with the implementation process, including developing intricate care pathways, collaborating with multiple disciplines and services, supporting and advocating for our patients and families, and interacting with government agencies and pharmaceutical companies. Case studies will highlight the positive impact these treatments are making on the lives of children and adolescents with rare neurological disorders. Results: Spinal muscular atrophy and Neuronal Ceroid Lipofuscinosis Type 2 are both rare and devastating neurodegenerative conditions with significant morbidity and mortality. Health Canada and government funding agencies recently approved Nusinersen, Onasemnogene abeparvovec for the treatment of SMA and Cerliponase alfa for the treatment of CLN2, leading us to swiftly integrate these treatments into our standard of care. Conclusions: While implementing these novel therapies into clinical practice can be both challenging and rewarding, neuroscience nurses are positioned at the forefront to be leaders in this process at both organizational, national, and international levels.