06/04/2026
What if patients could unlock rare disease research by asking the right question up front? One patient community did.
The US-based International Rett Syndrome Foundation (IRSF) asked sponsors what it would take for them to invest in Rett syndrome research. After learning it would require resources like patient registries and scientific advisory infrastructure, the Rett community built those resources from the ground up.
Now, there are 25+ companies researching Rett syndrome treatments.
In an article from Pink Sheet, Gvantsa Kvantaliani, Associate Director, Rare Diseases at Parexel, founder of Georgia's first patient-led rare disease organization and board member for Rett Syndrome Europe, spoke about the profound impact of patient involvement saying, "Such examples move the discussion beyond calls to 'letโs please involve' patient advocates in drug research to recognizing how they can actively drive progress."
Click through below to hear more from Gvantsa on the work IRSF has done and what challenges remain.๐โฌ๏ธ