DRF funds CRISPR research to correct Duchenne mutations

DRF funds CRISPR research to correct Duchenne mutations

The Duchenne Research Fund has awarded a £200,000 grant to the team at the Hospital for Sick Children in Toronto, Canada (‘Sick Kids’), who are working on using CRISPR/Cas9 technology to correct duplication mutations for Duchenne. Sick Kids’ incoming President and CEO...

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...

Solid Biosciences news

Solid Biosciences recently reported that it has raised up to $50 million which will enable the biotech to continue to advance its programs for Duchenne muscular dystrophy, including development of a microdystrophin gene. We are forever grateful to our incredible...

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-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 –...