Question to the Department of Health and Social Care:

To ask the Secretary of State for Health and Social Care, what steps his Department is taking to ensure that patients with Duchenne Muscular Dystrophy have access to new treatments in the NHS.

NHS England is responsible for commissioning specialised neurological services, including services for patients with neuromuscular disorders such as Duchenne Muscular Dystrophy. NHS England is working with the Neurological Alliance in support of the new national Neurology Advisory Group, which is considering ways to reduce variation and drive improvement in neurological care. Specialised neuromuscular care may include referral to local physiotherapy, hydrotherapy or rehabilitation if appropriate.

Further to this, the Royal College of General Practitioners has developed a learning module with Muscular Dystrophy UK to help general practitioners have a better understanding of their role in the management of neuromuscular conditions. It will also help them recognise the key moments when a patient needs to be referred to a specialist neuromuscular service.

The National Institute of Care and Health and Care Excellence is currently developing guidance on the use of eteplirsen (Exondys 51) for Duchenne Muscular Dystrophy through its highly specialised technology evaluation programme.