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Fordadistrogene movaparvovec

Fordadistrogene movaparvovec is a gene therapy treatment for people with Duchenne muscular dystrophy, manufactured by the pharmaceutical company Pfizer.

What stage is it at?

It is currently in the early steps in the NICE appraisal process. The timelines for the next steps are currently unknown and more detail will be available in due course. 

What are we doing?

MDUK will be monitoring for any further developments and feed into the appraisal when it opens.   

For more information on NICE’s assessment of Fordadistrogene movaparvovec can be found here. 

Support and information

Duchenne muscular dystrophy

Duchenne muscular dystrophy is a muscle wasting condition caused by the lack of a protein called dystrophin. It usually affects only boys.

Research

We fund groundbreaking research to learn more about muscle wasting conditions and lead us to new treatments. We’ve already made advances that would have been unthinkable just 10 years ago, and we are determined to go even further and faster.

Our support services

We are here for everyone, but we understand that support isn’t one-size-fits-all. Speak to us. We can tailor our support to meet your unique needs.

Our helpline is open Monday-Thursday 10am-2pm.