Rare Disease Day observed on 28 February 2026 with the theme More Than You Can Imagine

Key facts

• Rare Disease Day 2026 is observed globally on 28 February with the official theme More Than You Can Imagine.
• Approximately 300 million people worldwide live with a rare disease, of which nearly 80 per cent are genetic and 70 per cent appear in childhood.
• Statistics indicate that 95 per cent of rare diseases have no approved therapy, and the average time to receive an accurate diagnosis is 4.8 years.
• Nearly 30 per cent of children affected by rare diseases do not survive beyond their fifth birthday.
• The year 2026 marks the tenth anniversary of the National Alliance for Rare Diseases Support – Malta, founded by Michelle Muscat.
• UK-based Healx is applying Artificial Intelligence (AI) to identify drug repurposing opportunities for conditions such as Fragile X syndrome.
• Italian biotech AAVantgarde Bio is developing treatments for Usher syndrome type 1B by splitting and reassembling large genes inside the body.
• Beam Therapeutics is advancing base editing for Alpha-1 antitrypsin deficiency, while Tune Therapeutics is focusing on epigenetic tuning.
• Actio Biosciences is developing therapies for KCNT1-related epilepsy, and French firm InnoSkel is targeting Type II collagenopathies.
• AstraZeneca reinforced its rare disease strategy through the acquisition of Alexion Pharmaceuticals.
• India link not mentioned in the article.