BRC/NIHR GOSH Gene Therapy in Paediatrics

Today, 28th January 2026 we were invited the BRC/NIHR GOSH Gene Therapy in Paediatrics event to represent our small but determined community.

The day focused on bringing together GPs, paediatricians, clinicians and patient advocacy groups to provide an understanding of gene therapy technologies, long term effects, latest advancements & the future of gene therapy. 

For us, the most impactful part of the day was hearing from the patients and their families who’ve received gene therapy and the incredible benefits that have been truly life-changing 🩷💙

We’re grateful to be part of these conversations and to ensure BPAN families are seen, heard and represented. We even bumped into one of our own BPAN Gene Therapy Professors Simon Waddington!