In close collaboration with our sister non-profit organizations A Cure for Sophia and Friends in the United States (founded in 2023) and A Cure for Sienna in Australia (founded in 2022), we’ve fundraised 600,000 € (as of June 2026) and are supporting seven research and drug development programs.  

In alignment with our scientific advisors we perform research studies to better understand the underlying disease mechanism and unravel this complex neurodevelopmental disorder. Based on this pivotal work we derived two pathways for a treatment:  

1. Drug repurposing studies in mouse models and a patient iPSC derived cell model for treatment of the neurological symptoms of MCOPS12  
2. A disease-modifying RNA therapy (antisense oligonucleotide, ASO) to tackle the genetic root cause of MCOPS12 and improve neurological symptoms.