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Rare Disease case study

Last updated: May 7, 2025

Over 90% of rare diseases have no treatment… 

  • There are more than 6,000 known rare diseases…
  • Approximately 80% of them have a genetic component… 75% affect children…
  • And most rare diseases are chronic conditions, many of which progress overtime and are potentially fatal…

Understanding the rare disease framework is key to be able to offer study participants adapted solutions and as such, positively contribute to important and necessary clinical research conducted on rare diseases.

Kayentis offers an extensive experience in rare disease clinical trials, in indications such as:

  • Prader-Willi Syndrome
  • PIK3CA-related overgrowth spectrum
  • Auto-Immune Hepatitis (AIH)
  • Hypoparathyroidism
  • Sjögren’s syndrome
  • Acute Vertigo

Download the case study 👇
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