08/05/2026
Well this is wonderful news for the Duchenne community but bittersweet for me as sadly the National Institute for Health and Care Excellence (NICE) have decided NOT to approve the drug for non-ambulant, so all those that can’t walk like me are left out…again! This decision has taken NICE 2 years…2 years of damn hard graft from those involved in the Duchenne world…Where is the inclusivity NICE or is it simply about cost £££?!! Where is the hope for the non-ambulant population living everyday with Duchenne knowing our arms, our hearts etc etc are getting weaker and weaker…yet you decide to deprive access to a now approved drug to slow this awful disease down! Shocking and sad! 😢
On a landmark day for our community, we are delighted to share that the National Institute for Health and Care Excellence (NICE) has recommended givinostat for use on the NHS in England. In final draft guidance issued today, NICE approved the treatment for people with Duchenne muscular dystrophy who are ambulant (able to walk or stand), aged six and older.
After a long and challenging NICE appraisal process that lasted almost two years, and 18 months after the treatment was approved as safe and effective by the Medicines and Healthcare products regulatory agency, this marks a pivotal moment for people with Duchenne.
Today’s news offers real hope for children and young people with Duchenne and their families. It is the culmination of 14 years of work by Duchenne UK, our partners and other organisations, helping to drive advances in research, expand clinical trials and accelerate access to new treatments in the UK.
You can read more about this decision, and what it means, here: duchenneuk.org/givinostat-approved-for-use-in-nhs-england/