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To ask Her Majesty's Government what public funding is currently available for the development of exon skipping and other treatments for Duchenne muscular dystrophy; and what assessment they have made of the efficacy and promise of exon skipping in the treatment of muscular dystrophy.
The results of the recent United Kingdom exon skipping clinical trial, on patients with Duchenne muscular dystrophy, confirm that dystrophin was being produced, and was working correctly in muscles. However, the response was very variable between patients.
The drug tested in this clinical trial was developed by scientists in the MDEX consortium and they conducted this trial with funding from the UK Medical Research Council and the pharmaceutical industry.
The Medical Research Council provides funding for research through a range of grants and personal awards to scientists in universities, medical schools and other research institutes. They do not have set budgets for specific illnesses. Research proposals in all areas compete for the funding available and research excellence is the primary consideration in funding decisions.
We are unable to assess how effective this potential new treatment will be at slowing the progression of Duchenne muscular dystrophy. Larger and longer clinical trials will be needed to determine this. These trials are now being planned.