• Precision medicine: Personalized treatment approaches based on an individual's genetic and molecular profile could lead to more effective therapies tailored to the specific characteristics of their HD. Looking ahead, continued investment in rare disease research, infrastructure, and policy initiatives is critical for overcoming existing challenges and maximizing the potential of scientific advancements to improve the lives of individuals with rare diseases. Multidisciplinary collaborations and inclusive research practices are needed to effectively address the complex and evolving landscape of rare diseases.