World first for Leeds as drug is prescribed

A potentially life-changing drug is being prescribed for the first time for people with a rare genetic condition by specialists in the city.

Monday, 26th February 2018, 8:27 am
Updated Monday, 26th February 2018, 8:30 am
SPECIALISTS: The team at Leeds Teaching Hospitals NHS Trust.

A specialist centre at St James’s University Hospital has become the first place in the world to prescribe the drug Idebenone for some patients with Duchenne muscular dystrophy.

Studies have suggested the medication can slow down the deterioration of respiratory function in people with the condition, meaning they may not have to rely on breathing equipment as early in their lives. It is the first time Idebenone has been used for DMD outside a clinical trials setting.

Idebenone is being made available at the Leeds Sleep and Non Invasive Ventilation service as part of a government scheme set up to give patients with debilitating conditions access to promising new medicines sooner. Dipansu Ghosh, Consultant Respiratory Physician said: “Usually most of these patients would need ventilatory support by their early teens, which has huge implications for them and their carers.

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“Idebenone is a drug that has been shown to slow down the decline of respiratory function for patients with Duchenne Muscular Dystrophy and it will be prescribed as part of MHRA Early Access to Medicines scheme (EAMS).”

The EAMS scheme is designed to make drugs available where not enough data on their effectiveness has been collected to have them a approved by the National Institute for Health and Care Excellence (NICE).

Idebenone works by increasing the production of energy by mitochondria, and is already used to treat other conditions.

A spokesperson for Leeds Teaching Hospitals NHS Trust added: “This is the first time the drug has been prescribed outside a clinical trial setting and, if it works as the studies suggest, then it will delay the decline of lung function for these patients and might delay the need to have support with ventilation. Duchenne muscular dystrophy is a genetic disorder characterised by progressive muscle degeneration and weakness.”

The Sleep and Non Invasive Ventilation Service has been providing sleep and home ventilation services for patients since 1993. It is part of the Leeds Centre for Respiratory Medicine. The team operate a tertiary referral centre for complex ventilation across large parts of Yorkshire and Lancashire.

They care for around 4,500 patients who use continuous positive airway pressure (CPAP) machines and about 1,100 who are on home ventilation. The centre looks after around 27 people who have DMD and around eight are being prescribed the new drug.


Muscular dystrophies are a group of inherited genetic conditions which cause the weakening of muscles and increasing levels of disability.

Some types of affect the heart or the muscles used for breathing, at which point the condition becomes life-threatening. Duchenne muscular dystrophy is said to be one of the most common and severe forms of the condition.

It usually affects boys in their early childhood and men with the condition usually live into their 20s 30s.