In March 2017, Action Duchenne announced our funding of “UNITE-DMD”, one of the first gene therapy trials for Duchenne muscular dystrophy here in the UK.

From the very early days of Action Duchenne, we have supported research into gene therapy as a potential way of treating Duchenne muscular dystrophy. This area has seen significant progress in recent years, and now is the time to test its safety in people living with Duchenne, here in the UK.

This international collaboration will assess the safety of gene therapy in Duchenne, in a phase I/II clinical trial that will take place in both the UK and France. As these will be the first gene therapy trials for Duchenne in the UK, UNITE-DMD will be vital to test the safety of this technology.

Together with the principal funder Muscular Dystrophy UK, we are investing over £1.6m into UNITE-DMD. The French Muscular Dystrophy Association (AFM-Téléthon) is funding the French arm of the project.

Click here for full article about UNITE-DMD