2nd European Congress on Rare Diseases and Orphan Drugs

The 2nd European Congress on Rare Diseases and Orphan Drugs addresses the current landscape of rare diseases and orphan drug development in Europe.

Key Topics

• Public health perspectives in rare diseases
• Rare skin diseases
• Novel therapies for rare diseases
• Epidemiology and disease control
• Cardiovascular & respiratory rare disorders
• Functional genomics & gene therapy for rare diseases
• Approaches to rare disease diagnosis
• Rare hepatic and renal disorders
• Drug repurposing
• Orphan drug pricing and market dynamics
• Ethical & regulatory challenges
• Precision and personalized medicine
• Rare endocrine and neurological disorders
• Orphan drug development & challenges
• Newborn screening & rare pediatric disorders
• Cystic fibrosis
• AI in rare diseases and orphan drugs
• Rare cancers & drug development
• Genetic & hematologic rare disorders
• Biomedical research
• Advances in rare disease research
• COVID-driven rare insights
• Rare diseases and orphan drugs
• Biomarker importance in rare diseases

Who should Attend

• Regulators & policymakers
• Pharmaceutical & biotechnology professionals
• Rare disease community
• Patient advocates
• Academic experts
• Healthcare professionals
• Researchers
• Industrial stakeholders
• Market access consultants