Post by Parveen D.

Rare Disease Day 2025 ✨🧠 I was honoured to take part in Rare Disease Day this year, once again working alongside the amazing Dr Louise C. Gregory PhD and Federica Buonocore to raise awareness about paediatric pituitary disorders and the importance of ongoing research in this field. This marks my second time supporting the event as a GOSH YPAG (Young Persons Advisory Group) member and each year reinforces the critical role of effective scientific communication, public outreach and the importance of research into rare diseases. At our stall, we engaged young visitors through a range of interactive activities designed to spark curiosity and deepen understanding: 🧪 Micropipetting demonstrations—allowing children to try a fundamental lab technique used in medical research 🧠 3D brain model exploration—helping visualise neuroendocrine functions and the role of the pituitary gland 📖 Educational worksheets—highlighting the endocrine system’s impact on the body and its connection to rare diseases Beyond the stall, I had the opportunity to engage with researchers and learn about some groundbreaking work in the field of paediatric medicine, including: 🍽️ The ketogenic diet as a medical treatment for epilepsy—offering a potential non-pharmacological approach to seizure management 🧬 Nanotechnology-based drug delivery for neuroblastoma—aiming to enhance the specificity of retinoic acid therapy, reducing side effects and improving treatment outcomes. Rare Disease Day serves as a powerful reminder of the importance of continued research, collaboration, and outreach in improving patient outcomes. Grateful to have been part of such an inspiring event! One of the most rewarding moments of the day was finally meeting fellow members of the Great Ormond Street Hospital Young Persons’ Advisory Group in person for the first time since our meetings transitioned online. After years of connecting virtually, it was incredibly valuable to talk face-to-face, and strengthen the sense of collaboration that has been so central to our work in advocating for young patients and improving paediatric research initiatives. And last but definitely not least, a massive thank you to Deirdre Leyden who invited me to get involved with the event again this year! Events like these reinforce the importance of research, collaboration, and science communication in advancing paediatric healthcare. Grateful to be involved! #RareDiseaseDay #MedicalResearch #Paediatrics #ScienceCommunication #Endocrinology #GOSH #RareDiseaseDayGOSH