Muscular Dystrophy: Health Services

The Government is committed to improving the lives of those living with rare diseases, such as Duchenne muscular dystrophy (DMD). The UK Rare Diseases Framework sets out four priorities collaboratively developed with the rare disease community, and these include better coordination of care, and improving access to specialist care, treatment, and drugs. We remain committed to delivering under the framework, and will publish an annual England action plan in 2025 which will report on progress.

The National Institute for Health and Care Excellence (NICE) makes recommendations for the National Health Service on whether new licensed medicines should be routinely funded by the NHS, based on an assessment of their costs and benefits. The NHS in England is legally required to fund medicines recommended by the NICE, normally within three months of the publication of final guidance. The NICE has recently published guidance recommending vamorolone as an option for treating DMD in people four years old and over, and is currently developing recommendations on the use of givinostat, which was granted a conditional marketing authorisation by the Medicines and Healthcare products Regulatory Agency for the treatment of DMD in patients aged six years old and over in December 2024.