New NHS treatment for Duchenne muscular dystrophy

New NHS treatment approved for Duchenne muscular dystrophy after NICE agreement

Hundreds of children and young people in England living with Duchenne muscular dystrophy (DMD) are set to gain access to a newly approved treatment after a commercial agreement cleared the way for NHS funding.

The National Institute for Health and Care Excellence (NICE) has approved givinostat (brand name Duvyzat), a treatment designed to slow the progression of Duchenne muscular dystrophy, a rare inherited condition that causes progressive muscle weakness and loss of muscle function over time.

DMD primarily affects boys and usually becomes apparent in early childhood. As the condition progresses it can increasingly affect mobility, independence, heart function and breathing.

 

Funding available immediately

NICE said givinostat will be funded immediately through the NHS Innovative Medicines Fund following a commercial deal with NHS England. The organisation described it as an important additional option for people with Duchenne, noting that the treatment works regardless of the specific genetic variant responsible for the condition.

The treatment has already been available to some patients through an Early Access Programme, but the latest decision means wider NHS availability for eligible patients in England.
 

Maintaining muscle function for longer

Clinical studies suggest givinostat can help slow the decline in muscle function and may help people maintain mobility for longer. Unlike some Duchenne treatments that target particular genetic mutations, givinostat may benefit a broader group of patients because it addresses processes linked to inflammation and muscle damage.

Current guidance applies to people aged six and over who are still able to walk or stand at the beginning of treatment and who meet the relevant clinical criteria. Around 530 people in England are expected to benefit.
 

No cure, but treatment slows progression

Patient organisations welcomed the announcement. Duchenne UK said the decision represented important progress for families affected by the condition and highlighted years of campaigning work aimed at improving access to new treatments.

While givinostat is not a cure for Duchenne muscular dystrophy, campaigners and clinicians hope that slowing disease progression and helping people maintain movement for longer could make a meaningful difference to everyday life and independence.
 

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Further reading and resources

There is more about the condition and the latest research on Duchenne UK (external link will open in a new browser tab or window)

We have an area of Independent Living with products to help families and carers of a disabled child

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Guest blog: Living independently with muscular dystrophy

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