Vamorolone receives Fast Track designation from FDA

We’re pleased to share news from ReveraGen, the company developing the novel steroid alternative Vamorolone (previously called VBP15), that their drug has received Fast Track designation from the FDA, which will speed up the review of Vamorolone. In 2014, Duchenne...

ReveraGen BioPharma licenses option to develop Vamorolone with Actelion

We’re sharing news that ReveraGen BioPharma has licensed an option to develop Vamorolone with Actelion Ltd. Vamorolone is the first in-human steroid like compound that has shown in animal models to have many of the benefits of corticosteroids, without the many side...

Duchenne patients recruited for steroid replacement study

We are delighted to announce that ReveraGen BioPharma, the company developing a steroid replacement for Duchenne muscular dystrophy, has begun recruiting patients for its Phase 2a open label study. The trial will be recruiting patients who haven’t yet started...
DRF raises £1.2 million to help beat Duchenne

DRF raises £1.2 million to help beat Duchenne

The Duchenne Research Fund raised £1.2 million at its ‘The Time Is Now’ Gala Dinner on 5 May at the Lancaster London Hotel to help find a cure for Duchenne muscular dystrophy – the biggest genetic killer of children worldwide. The funds raised will enable the DRF to...

DRF-funded drug development programme receives further £4.6 million

The Duchenne Research Fund, the Duchenne Children’s Trust and Joining Jack and are delighted with the news from the John Walton Muscular Dystrophy Research Centre that they have received £4.6 million in European funding for a Phase II clinical trial for Vamorolone –...
Dog with dystrophin mutation saved from Duchenne

Dog with dystrophin mutation saved from Duchenne

DRF-funded research was published this week in Cell, one of the world’s most prestigious scientific journals, analysing why two golden retrievers showed no severe symptoms of Duchenne muscular dystrophy and unexpectedly lived a normal lifespan despite being born...

Gene therapy breakthrough in muscular dystrophy dogs

The Duchenne Research Fund is incredibly proud of Dr Dongsheng Duan – an important Scientific Advisory Board member of Solid GT, a gene therapy programme pioneered by Solid Biosciences and that we helped to fund. Dr Duan’s latest gene therapy work on...

DRF-funded study shows encouraging results in combating muscle-wasting

Professor Dame Kay Davies (University of Oxford) and her group have recently published encouraging results of a study to test new second-generation compounds to increase the level of utrophin protein as a treatment for boys and men with Duchenne and Becker muscular...

The Duchenne Research Fund partners with Solid Biosciences

With the funds we raised at our 2013 Gala Dinner we brokered the first partnership between UK Duchenne charities and commercial enterprise to launch Solid GT, an exciting gene therapy project. We approached US-based Solid Biosciences to present promising gene therapy...