Dr Emily Whitehouse is the latest consultant to join the fight against DMD at the Royal Manchester Children’s Hospital   The DMD Hub is Duchenne UK’s programme to expand capacity for Duchenne muscular dystrophy (DMD) clinical trials in the UK. The DMD Hub has funded 30 key posts at hospital sites in the UK to-date, from research nurses to clinical trial coordinators, allowing many more boys to take part in clinical trials for potentially life-changing treatments.    We’re pleased to announce that Dr Emily Whitehouse, who was initially appo ..read more

Breaking News: Duchenne UK backed drug shows success in clinical trials Duchenne UK is delighted to announce that Vamorolone, a drug to treat Duchenne muscular dystrophy (DMD), has shown positive results in a Phase 2 Study. Vamorolone was developed as an alternative medicine to corticosteroids, that offers the benefits of steroids, but without some of the side effects that can have a serious impact on patients’ quality of life. The results from Santhera Pharmaceuticals and ReveraGen BioPharma showed that boys on Vamorolone did better than those taking a placebo. It also found that Vamorolone h ..read more

19 May 2021 Duchenne UK is committed to the ongoing success of the DMD Hub and today announced a further four years of funding for the DMD Hub Manager role, bringing total investment in the DMD Hub to £4.3million.   The DMD Hub is one of Duchenne UK’s flagship projects and has successfully expanded capacity for clinical trials for Duchenne muscular dystrophy (DMD) treatments in the UK. In the first four years of the project, Duchenne UK and its partner charities invested £2.7m in the DMD Hub, with an additional £1.6m committed over the next 4 years.   The DMD Hub is a colla ..read more

Pfizer have announced the enrolment of their first participant in a Duchenne muscular dystrophy (DMD) gene therapy trial in the UK. This is the first time a DMD patient in the UK has received gene therapy, and is a landmark moment in the mission to treat the condition. The patient was enrolled at Newcastle, one of three locations in the UK where the DMD C3391003 Gene Therapy study is taking place. For more details on the trial in the UK, please consult the DMD Hub's Clinical Trial Finder here. The trial is a Phase 3 global, multicentre, randomised, double-blind, placebo-controlled study. In to ..read more

12 May 2021 Duchenne UK have funded a study that aims to improve our knowledge of treating heart muscle disease in patients with Duchenne muscular dystrophy (DMD).  Almost all people with DMD will develop heart muscle disease, or cardiomyopathy, at some stage. There are drugs that are believed to slow down damage and protect the heart. However, no clear data exists for their effective use in DMD.   In 2011, a clinical trial, called The Heart Protection Study, was set up to see if starting two existing heart medications in co ..read more

Pharmaceutical companies Santhera Pharmaceuticals and ReveraGen BioPharma have announced new data from their clinical study of a potential treatment for Duchenne muscular dystrophy (DMD) that could replace the current standard of care, corticosteroids. Their investigational drug, Vamorolone, has the potential to slow disease progression in DMD in a similar way to corticosteroids, but with less severe side effects. Vamorolone was developed as a dissociative steroid, meaning that it may be able to chemically separate the clinical benefits of steroids from their safety concerns. Duchenne UK and o ..read more

6 May 2021  DMD Care UK have launched the first comprehensive patient survey aiming to understand Duchenne muscular dystrophy (DMD) patients’ and caregivers’ experiences of medical care.   DMD Care UK, a joint initiative funded by Duchenne UK, Joining Jack and the Duchenne Research Fund, aims to ensure that everyone living with DMD has access to the best care. In collaboration with the John Walton Muscular Dystrophy Centre and the North Star Network, the project will bring together exp ..read more

5 May 2021 Duchenne UK has invested £287,500 into a new 18-month study investigating whether Lipid Nanoparticles (LNPs) could be used to help in gene therapy treatments for DMD.   Lipids are naturally occurring small fatty molecules that exist within the body, and nanoparticles refers to their small size. LNPs are currently being used as a key component in the Pfizer/BioNTech and Moderna COVID-19 vaccine.  Duchenne UK is now funding work to see whether these LNPs could be used to deliver gene therapy in DMD.   Several clinical tr ..read more

26 July 2018 Sarepta have been asked by the FDA to put on temporary hold their clinical trial looking at the safety and efficacy of their gene therapy treatment. In an open letter to the community, Chief Exec of Sarepta Therapeutics explained the Phase 1/2a clinical trial has been put on hold due to an ‘out-of-specification production lot’. To understand what this means, we need to know a little about how materials for administration of the drug are produced for the trial. Sarepta produce the actual gene therapy material - the ‘active’ part of the drug.However, this can’t be given straight to ..read more

4 May 2018 Last week, at the European Medicine’s Agency (EMA), the Committee for Medicinal Products for Human Use (CHMP) held a hearing looking at whether to grant a regulatory approval for eteplirsen (also known as Exondys 51), in Europe. Eteplirsen is designed to treat an estimated 13% of the Duchenne population amenable to the skipping of exon 51. We are disappointed to share the news that the CHMP’s trend vote was negative. This means that efforts to bring eteplirsen to people living with Duchenne in Europe will be delayed. You can read more in this ..read more