Rare diseases persist in presenting notable challenges for individuals, families, and the healthcare community. This conference serves as an outstanding platform to exchange knowledge, share experiences, and cultivate collaborations in addressing these challenges. Our goal is to assemble global experts, advocating for innovative approaches, progressing scientific research, and elevating the standard of care and support available to those with rare diseases.
Topics: Rare Diseases - The big picture, Basic Diagnostic Approaches to Rare Disease, Treatable Rare Disease, Focus on Therapies for Rare Diseases, Treatable Genetics Disorders: An Update, and Prevention