
I know all too well from my own experience caring for our dear son, Ivan, that for millions of patients living with rare diseases and their families, time matters. Too often patients and their loved-ones face years of uncertainty, worry and heartache, with few or no treatment options available to them.
Today’s announcement from @MHRAgovuk is an important and hopeful step towards changing that, while helping the UK become a powerhouse of drug development for genetic and rare diseases. By recognising the unique challenges of developing and delivering rare disease treatments to patients, and the need for an innovative, nimble and adaptable regulatory approach, the UK is showing real international leadership, building on the success of @GenomicsEngland and the 100k Genomes Project, both of which I launched as Prime Minister.
Through my work with the @OHRareDisease Centre, we see every day the extraordinary potential of scientific innovation to transform lives. What patients and families need now is a system that can move with the same urgency as the science.
MHRAgovuk@MHRAgovuk
Fewer than 5% of rare diseases have an approved treatment. The average diagnosis takes over five years. We're proposing a new way forward, and we want to hear from patients, families and carers like you. Open to all, respond before 30th July 2026 – brnw.ch/21x2GHn
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