Nearly £750,000 invested for new Duchenne muscular dystrophy research

Published Date
Dr Özge Özkaya

We are delighted to announce that we will be funding new research projects into Duchenne muscular dystrophy commencing later this year. The projects reflect our commitment to accelerating the pace of developing effective treatments for Duchenne muscular dystrophy.

Scientific research into Duchenne muscular dystrophy is now at a critical stage with many approaches already being tested in clinical trials.

The projects will mostly focus on therapeutic approaches to tackle the condition and will use cutting edge molecular biology techniques such as gene therapy and genome editing.

We are very pleased to support the on-going work in Professor Kay Davies and Professor George Dickson’s laboratories, both developing second-generation compounds/therapies for the treatment of Duchenne muscular dystrophy. We are also excited to be funding research in areas that are understudied such as the effect of Duchenne muscular dystrophy on the brain.

The projects were selected following a thorough peer review process using independent international experts as well as our Lay Research Panel, who are affected directly or indirectly by a muscle-wasting condition and deemed the research to be relevant to people living with muscle-wasting conditions. Read more about how we fund research.

Here are the projects that we will be funding:

Professor Kay Davies – Identifying small molecules for Duchenne muscular dystrophy

Professor George Dickson – Moving closer to a gene therapy for Duchenne muscular dystrophy

Professor Francesco Muntoni and Dr Francesco Conti – Genome editing to repair duplications in Duchenne muscular dystrophy

Professor Volker Straub – Investigating the effect of Duchenne muscular dystrophy on the brain

We were able to fund the research projects thanks to the support of UK families and other donors who support the Duchenne Research Breakthrough Fund (DRBF). The aims of the DRBF are to fund the very best scientific research into Duchenne muscular dystrophy, to ensure the development of vital infrastructure for clinical trial readiness and to work with regulatory bodies and pharmaceutical companies to ensure patients have early access to emerging treatments.

One of the projects is funded in partnership with the Duchenne Children’s Trust.

It is only through your contributions that we can continue to fund the vital work that takes us closer to finding treatments and cures for muscle disease. Donate now and help change the lives of thousands of people living with muscle disease. Thank you for your support.

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