Watch the highlights of An Evening with the DRF

Watch the highlights of Gavriel Rosenfeld in conversation with Dr Ronni Cohn at our virtual event: An Evening with the Duchenne Research Fund. More than 400 people tuned in to hear Gavriel talk about living with Duchenne, mental health, juggling school and hospital...

Solid announces collaboration with Ultragenyx

Ultragenyx Pharmaceutical and Solid Biosciences have announced a strategic collaboration and license agreement to focus on the development and commercialisation of new gene therapies for Duchenne. The collaboration brings together Solid’s expertise in muscle biology...

Update on Ignite DMD from Solid Biosciences

Solid Biosciences has issued an update on IGNITE DMD, the Phase I/II clinical study with its microdystrophin gene therapy, SGT-001. The most recent child dosed in the study has experienced a serious adverse event (SAE), and thankfully, he is now improving. He is being...

Gene therapy for Duchenne: latest news

Five years ago we identified gene therapy as a viable treatment for Duchenne. Today we are reflecting on the exciting and inspiring events of the past week in the gene therapy arena which are now bringing hope and cautious optimism to the Duchenne community. We have...

Solid Biosciences clear to resume IGNITE DMD

Message from Solid Biosciences: “We’re pleased to share with you that the FDA has lifted the clinical hold on IGNITE DMD, our clinical trial investigating SGT-001 as a potential treatment for Duchenne muscular dystrophy (DMD). SGT-001 is a microdystrophin gene...
DRF’s funding of Solid GT leads to $42.5 million investment

DRF’s funding of Solid GT leads to $42.5 million investment

We are thrilled that the money raised through the Duchenne Research Fund’s 2013 dinner and donations since then have enabled us to seed fund the incredible Solid GT project, which has today received industry investment (read the press release below). While gene...
Albourne raises £155,000 for DRF

Albourne raises £155,000 for DRF

We are immensely grateful to Albourne Partners and their supporters for raising more than £155,000 for our projects at their incredible performance of ‘The Importance of Being Lewis’ on 7 October. The event was held to honour the memory of Jacques Joubert...

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...