Abstract Topics

  • General challenges in rare disease
  • Patients focused aspects in rare diseases
  • Rare syndromes
  • Single gene diseases
  • Relations between rare diseases and common disorders
  • Miscellaneous rare diseases
  • New treatment modalities
  • Role of registries and big data
  • Ethical aspects in rare diseases
  • Animal models for drug development for rare diseases
  • Lysosomal storage disorders
  • Genetic Parkinson’s disease