Question to the Department of Health and Social Care:

To ask the Secretary of State for Health and Social Care, what recent steps the Government and NHS have taken to improve research, treatment and outcomes for Duchenne muscular dystrophy.

The Government published the new United Kingdom Rare Diseases Framework in January 2021, outlining the key priorities for rare diseases including improving access to specialist care, treatments and drugs. The National Institute for Health and Care Excellence has published guidance recommending ataluren for the treatment of eligible patients with Duchenne muscular dystrophy, subject to a managed access agreement which has recently been extended from July 2021 to January 2023.

Since 2018, the National Institute for Health Research has directly funded four studies relating to Duchenne Muscular Dystrophy in addition to providing infrastructure support to 86 studies.