SAPS-056 - Bridging the gap to innovative therapies for pediatric rare diseases: The role of clinical trials

• At: Copenhagen (Denmark) (2025)
• Type: Poster
• Poster code: SAPS-056
• By: STOYNOVA, Hristina (Department of Organisation and Economics of Pharmacy, Faculty of Pharmacy, Medical University of Plovdiv, Bulgaria)
• Co-author(s): Mrs Hristina Stoynova (Department of Organisation and Economics of Pharmacy, Faculty of Pharmacy, Medical University of Plovdiv, Plovdiv, Bulgaria)
  Radiana Staynova (Department of Organisation and Economics of Pharmacy, Faculty of Pharmacy, Medical University of Plovdiv, Plovdiv, Bulgaria)
  Assoc. Prof. Daniela Kafalova (Department of Organisation and Economics of Pharmacy, Faculty of Pharmacy, Medical University of Plovdiv, Plovdiv, Bulgaria)
  
• Abstract:

  Background
  According to the European Commission, rare diseases affect approximately 6–8% of the global population, with a significant proportion manifesting in childhood. These diseases are typically chronic or progressive and can lead to severe health complications. While individual rare diseases are uncommon, collectively, they impact a large..

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