NEURO NEWS

Every year on the last day of February, the world turns its gaze toward a community that is often invisible, yet 300 million strong. February 28, 2026, marks the 19th annual Rare Disease Day.

The 2026 theme, "More Than You Can Imagine," is a powerful reminder that while a single disease may be rare, the collective experience of living with one is a global reality. This year, the focus is squarely on Equity—ensuring that the "diagnostic odyssey" ends sooner and that life-saving treatments aren't restricted by geography or income.

The Reality of "Rare" in 2026

It’s a common misconception that rare diseases only affect a few. In reality:

• 3.5 million people in the UK alone live with a rare condition.

• 70% of these diseases start in childhood.

• 90% of rare diseases still have no European Medicines Agency or NICE -approved treatment.

However, 2026 is a year of significant momentum. In the UK, the Rare Diseases Framework has been extended through 2027, providing a vital bridge for policy and funding. Meanwhile, the ERDERA (European Rare Diseases Research Alliance) has launched major transnational calls to solve undiagnosed cases using AI and multi-omics.

Breakthroughs Worth Celebrating

The past year has seen "medical miracles" move from the lab to the clinic:

1. Personalised CRISPR: In a world-first, custom gene therapies are now being designed for single individuals, such as the landmark case of baby KJ Muldoon, whose life-threatening metabolic disorder was corrected with a bespoke genetic "pencil."

2. AI Diagnostics: New AI-driven tools are slashing the time it takes to identify rare variants, helping doctors spot patterns that used to take decades to uncover.

3. The "Plausible Mechanism" Pathway: Regulatory bodies like the FDA and MHRA are pioneering faster approval routes for ultra-rare conditions, prioritising biological evidence when traditional large-scale clinical trials are impossible.

A Future Built on Equity

Equity doesn't mean treating everyone the same; it means giving everyone what they need to thrive. Whether it's a child in a remote village getting a remote genomic consultation or a researcher finding a "plug-and-play" therapy for an ultra-rare cancer, the goal for 2026 is clear: Nobody left behind.

"Recognition is only the first step. Alongside awareness, we need concrete actions—clearer pathways, inclusive research, and services that are accessible to all." — Rhiannon Walls, Rare Disease Day Global Lead.

Therapies for Rare Diseases across the Neuro Therapy Network

Most of our Centres were initially set up to treat Multiple Sclerosis (MS). However, over the years, the range of conditions seen has increased to include other chronic and neurological conditions, including Parkinson’s, Stroke, and Cancer, Long Covid, ME/CFS and Fibromyalgia. Some Centres have seen some very rare conditions too, including: Ehlers-Danlos syndromes,  Labrune Syndrome, Hypokalaemia periodic paralysis, Guillain-Barré syndrome, Encephalitis and various forms of Ataxia.

To find an NTN Centre near you and to see what conditions they see and what therapies they offer, please visit www.neurotherapynetwork.org.uk/centres

For further information about Rare Disease Day 2026, please visit www.rarediseaseday.org