Duchenne muscular dystrophy Event

Last week I attended the Duchenne UK drop in session on Duchenne Muscular Dystrophy (DMD).

DMD is a severe, progressive muscle-wasting disease affecting approximately 2,500 people in the UK. It is caused by a genetic mutation that prevents the production of dystrophin, a protein essential for muscle function. DMD primarily affects boys, with symptoms usually appearing between ages 3-6. As the disease progresses, it leads to loss of mobility, respiratory difficulties, and heart problems. While life expectancy has improved, most individuals with DMD do not survive beyond their 30s.

Duchenne UK is the leading charity in the fight against DMD. Founded in 2012 by two mothers, Alex Johnson OBE and Emily Reuben OBE, whose sons were diagnosed with DMD, the charity has raised almost £30 million in accelerating research for treatments and a cure.

Givinostat, a promising new treatment for DMD, has been conditionally approved by the Medicines and Healthcare products Regulatory Agency (MHRA) for patients aged 6 and older.

An Early Access Programme (EAP) has been established to provide free access to givinostat while awaiting full NHS approval. However, systemic barriers within the NHS such as resourcing, administrative capacity, and fridge space, are preventing eligible patients from accessing this potentially life-changing treatment.

We need improved access to treatments, better funding for research, and increased awareness of this condition.

Let’s work together to break down the barriers preventing access to life-changing treatments through the Early Access Programme (EAP).