A lynchpin is the small but central fastener that holds a complex system together. In rare disease research and therapy development, patient advocates often serve this critical role, bringing together families, clinicians, researchers, and industry partners, and bridging gaps no other stakeholders can fill. This talk will highlight how advocacy contributes to the clinical trial ecosystem, supports access to approved and emerging therapies, and drives progress toward improved standards of care and patient outcomes. Practical examples will be shared from initiatives led by the Batten Disease Support, Research and Advocacy (BDSRA) organisations, including the recent story of Charleigh Pollock in Canada which illustrates the impact of global advocacy in securing access to life-changing therapy and influencing policy change.