With World Duchenne Awareness Day (7 September) just around the corner, we thought we’d whet your appetite by sharing the incredible progress that the Duchenne Research Breakthrough Fund (DRBF) has made!
The DRBF was launched in September 2012 as a way for families to support the translation of promising science from the laboratory into the clinic. This journey is made up of three parts, all of which the DRBF has significantly contributed to:
- Funding the best scientific research
The DRBF has funded a total of 28 research projects and one university lectureship. All of these came through Muscular Dystrophy UK’s rigorous peer review system, which ensures that the research and scientists we fund are of the highest quality.
- Investing in clinical trial infrastructure
To help make the UK ‘clinical trial ready’, the DRBF has invested in two clinical trial co-ordinator roles, the North Star database and three clinical roles that came out of the Newcastle plan.
- Ensuring access to treatments
The DRBF also supports campaigning activity related to Duchenne muscular dystrophy; this includes working with pharmaceutical companies and regulatory bodies to ensure that patients can access new treatments as quickly as possible. For example, funds raised through the DRBF were vital for the successful campaigning that led to Translarna being funded by NHS England.
To date, the DRBF has invested approximately £2.48 million into these areas. Of course, this wouldn’t have been possible without individual donations, family fundraising activities, trusts and corporate support. We want to say a huge thank you to all of those who have been involved and hope that we can continue to fight this devastating condition together.
Muscular Dystrophy UK will be announcing its new Duchenne muscular dystrophy research projects on World Duchenne Awareness Day – make sure to visit the website on 7 September to find out more! Thanks again to all the families and donors who are making this research happen.