What is the role of patients in the field of rare neurotransmitter diseases?

The first week of November was devoted to the International Neurotransmitter Disorders Conference, from 5th to 7th November. I was attending the 𝗜𝗻𝘁𝗲𝗿𝗻𝗮𝘁𝗶𝗼𝗻𝗮𝗹 𝗡𝗲𝘂𝗿𝗼𝘁𝗿𝗮𝗻𝘀𝗺𝗶𝘁𝘁𝗲𝗿 𝗗𝗶𝘀𝗼𝗿𝗱𝗲𝗿𝘀 𝗖𝗼𝗻𝗳𝗲𝗿𝗲𝗻𝗰𝗲 "𝘽𝙧𝙞𝙙𝙜𝙞𝙣𝙜 𝙎𝙘𝙞𝙚𝙣𝙩𝙞𝙛𝙞𝙘 𝙄𝙣𝙣𝙤𝙫𝙖𝙩𝙞𝙤𝙣 𝙖𝙣𝙙 𝘾𝙡𝙞𝙣𝙞𝙘𝙖𝙡 𝘼𝙙𝙫𝙖𝙣𝙘𝙚𝙨 𝙛𝙤𝙧 𝙋𝙖𝙩𝙞𝙚𝙣𝙩𝙨" as a representative of Hrabriša - Lil` Brave One.

This was the first of many posts about this conference, so you can read more, if you want, about this experience HERE.

Poster presentations

Poster presentations

We proudly presented our posters on the work of Hrabriša - Lil` Brave One, highlighting how patient-led efforts can bridge families, healthcare professionals, and researchers across borders.

Another key highlight was the development of Patient Reported Outcome Measures (#PROMs) for individuals with Rare Neurotransmitter Disorders (#RND), supported by the #iNTD network.

This initiative aims to better understand the real-life experiences of patients, families, and caregivers affected by neurotransmitter disorders.

By sharing their insights, participants will directly inform clinicians and researchers about what matters most in care and treatment — and help guide future research and clinical trials.

Here is more information about this topic: POSTER PRESENTATION

We gave voice to the patients

Patient Workshop

The Family Workshop, “𝗧𝗲𝗮𝗺𝘄𝗼𝗿𝗸 𝗳𝗼𝗿 𝗕𝗲𝘁𝘁𝗲𝗿 𝗢𝘂𝘁𝗰𝗼𝗺𝗲𝘀—𝗣𝗮𝘁𝗶𝗲𝗻𝘁-𝗟𝗲𝗱 𝗘𝗳𝗳𝗼𝗿𝘁𝘀 𝗶𝗻 𝗥𝗮𝗿𝗲 𝗡𝗲𝘂𝗿𝗼𝘁𝗿𝗮𝗻𝘀𝗺𝗶𝘁𝘁𝗲𝗿 𝗗𝗶𝘀𝗼𝗿𝗱𝗲𝗿𝘀”, at the 𝘐𝘯𝘵𝘦𝘳𝘯𝘢𝘵𝘪𝘰𝘯𝘢𝘭 𝘕𝘦𝘶𝘳𝘰𝘵𝘳𝘢𝘯𝘴𝘮𝘪𝘵𝘵𝘦𝘳 𝘊𝘰𝘯𝘧𝘦𝘳𝘦𝘯𝘤𝘦 in London brought together clinicians, researchers, and patient advocates who share one goal—improving lives through collaboration.

As a patient advocate, my focus has always been on bridging the gap between scientific discovery and lived experience. This workshop showed once again how important patient-led efforts are in driving meaningful progress in rare neurotransmitter disorders.

From early diagnosis and treatment pathways to psychosocial support, the patient voice is no longer an afterthought—it’s a catalyst for better outcomes.

I would like to extend my sincere thanks to the conference organisers, in particular Lisa Flint for creating this inclusive platform, and especially to Anna Pawlowicz, who guided our discussion with such insight and empathy.

Before "Goodbye"...

If you attended the conference or took part in the workshop, I’d love to connect and hear your impressions or takeaways. Continuing this dialogue beyond the event for Hrabriša - Lil` Brave One led by the amazing Ivana Badnjarević is how we ensure that collaboration doesn’t stop when the session ends.

Together, we move closer to transforming rare disease care from isolated efforts into shared impact. Now, let's see what is the next "mountain to move"?