Written by Ana Pataki, project coordinator, Hrabriša – Lil` Brave One

Do you have someone in mind when you hear the words “Lil’ Brave One”?

For us, it is, first and foremost, all the children living with conditions that change their lives and daily routines. But it is also their parents, siblings, friends, and everyone who shares their journey.

That is why our parent-led organisation, dedicated to the fight against rare congenital neurotransmitter disorders (RNDs), carries this very name—Lil’ Brave One (Hrabriša).

We come from Serbia, a country in Southeastern Europe, and we are the first non-EU patient organisation whose work has been recognised and supported by the Chan Zuckerberg Initiative through its invaluable “Rare as One” programme.

Like everywhere in the world, the number of people affected by this group of rare diseases is small—in Serbia, we are talking about around thirty families.

However, for these few dozen families, statistics do not matter, because their lives have been profoundly changed.

Our mission is to be a valuable support system for them. We want to improve diagnostics, enhance treatment opportunities for those affected by these complex conditions, and raise public awareness about these disorders. We strive to encourage the medical and scientific community to engage in ongoing research and contribute to the global knowledge pool.

Most importantly, we want patients to have access to adequate therapy, psychosocial support, comfort, understanding, and hope.

For strong scientific networks to support innovations in RNDs

Our primary goal is to contribute to a deeper understanding of neurotransmitter disorders within the scientific community. By connecting researchers, medical specialists, and families, we want to build a network that supports innovation and scientific advancements in rare diseases.

At this moment, our top priority is ensuring that the local and regional network of medical researchers stays up to date with the latest discoveries and active scientific projects in this field—and more importantly, that they actively participate in these studies.

Another key focus of ours is to strengthen the role and impact of the international working group on Neurotransmitter Related Disorders (iNTD), a global research project dedicated to primary and secondary neurotransmitter disorders.

We plan to develop a patient registry and support healthcare professionals in Serbia and beyond to join the iNTD network and include data on patients from our country and the region.

“With more data, we can support more research and provide detailed insights into diagnosis, treatment possibilities, and improved patient care. By connecting through research networks, we will facilitate collaboration among scientists focused on inherited metabolic disorders related to neurotransmitters, creating fertile ground for groundbreaking discoveries,”

Ivana Badnjarević, the founder of Lil’ Brave One, Serbia.

Through collaborative research networks, the organisation will connect scientists focused on neurotransmitter-related inborn errors of metabolism, nurturing an environment ripe for groundbreaking discoveries.

To support research and advocate for patients

Moreover, we plan to convene the disease community, hosting scientific gatherings that will unite stakeholders in shared purpose and passion. In the coming years, Lil’ Brave One will start with a strategic journey, filled with activities designed to uplift our RND community. From capacity-building initiatives that establish a strong Scientific Advisory board and executive team to the creation of young research forums and congresses, each step is carefully crafted to support patients and researchers.

Our organisation is committed to developing technical resources and platforms that will provide vital information and training, making the world of RNDs more accessible to everyone involved.

As Lil’ Brave One moves forward, its first year is planned to be a pivotal one.

The establishment of a dedicated management team and a Scientific Advisory Board will lay the groundwork for future successes. Formalising relationships with international networks, particularly the international working group on Neurotransmitter Related Disorder (iNTD), will facilitate vital scientific exchanges.

The RND Research Forum, a gathering of scientific community in the region,will serve as a beacon for emerging researchers, allowing them to share their findings and insights, and encouraging a spirit of collaboration and innovation.

As Lil’ Brave One strives to support research and advocate for patients, it carries with it the hopes and dreams of many. The dedication to transforming the landscape for those affected by RNDs is palpable, and the journey ahead is filled with promise.

Through collaboration, compassion, and unwavering commitment, we are determined to make a significant difference in the lives of countless individuals and families, lighting the way toward a brighter future.

Not only is the Lil’ Brave One organisation the only one funded by the Chan Zuckerberg Initiative in this part of the world but also stands among an elite group of only 30 grantees worldwide.

This recognition—alongside two other European organisations—underscores the organisation’s potential to elevate the voices of patients and families in the rare disease community.

For those interested in following this inspiring journey, we are inviting you to explore our website and join us in our mission to bring awareness, support, and hope to the rare disease community.

Check out our website and social media profiles on Instagram, pages on Facebook and Linkedin.

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