Funding to help sufferers of rare diseases November 5, 2014 Murdoch University researchers from the Centre for Comparative Genomics (CCG) have received almost $800,000 in funding from the National Health and Medical Research Council (NHMRC) to develop genetic drugs to treat rare diseases. Chief Investigator Professor Steve Wilton and Investigator Professor Sue Fletcher have previously had success in developing a therapy for people with Duchenne Muscular Dystrophy (DMD) and the new funding will allow them to extend their research to other rare conditions. Over the last decade Professor Wilton and Professor Fletcher developed a new drug for DMD sufferers that works by “skipping over” the faulty part of the gene message, so that is can produce a functional version of the protein dystrophin that stabilises the muscle fibre. “Clinical trials of the drug we developed for Duchenne Muscular Dystrophy have yielded some remarkable results,” Professor Wilton said. “We believe we can use the same technology that we applied in development of the drug for DMD to treat other rare diseases such as cystic fibrosis, spinal muscular atrophy and Pompe’s disease.” The (NHMRC) grant is worth $792,656 over four years starting in 2015. Also on the research team are Director of the CCG, Professor Matthew Bellgard and Director Genetic Services & Familial Cancer Program of WA, Professor Jack Goldblatt. Professor Fletcher said that it was crucial that universities researched drug development for rare diseases because the small number of patients requiring specific treatments is a disincentive to investment in therapy development. “When you add up all the people in Australia suffering from a rare disease there are at least 400,000 which is actually more than the number of people living with cancer,” Professor Fletcher said. “Yet despite the large number of people afflicted, there has been relatively little research on treatments for rare diseases. Within Australia, WA Health has been leading the drive to help those living with a rare disease and there is also increasing community awareness and advocacy, driven by community groups.” Professor Wilton and Professor Fletcher’s breakthrough treatment for DMD was recognized when they were awarded one of nation’s most prestigious awards last year, the Australian Museum Eureka Prizes, the NSW Health James Callachor Eureka Prize for Medical Research Translation. Print This Post Media contact: Hayley Mayne Tel: (08) 9360 2491 | Mobile: 0400 297 221 | Email: email@example.com Categories: General, Murdoch achievements, Health, biomedicine and psychology, School of Engineering and Information Technology Research Tags: australian museum eureka awards, centre for comparaticve genomics, cystic fibrosis, duchenne muscular dystrophy, national health and medical research council, nsw james callachor eureka prize for medical research translation, pompe's disease, spinal muscular atrophy, steve wilton, sue fletcher, wa health Leave a comment Name (required) Mail (will not be published) (required) Website You can use these tags : <a href="" title=""> <abbr title=""> <acronym title=""> <b> <blockquote cite=""> <cite> <code> <del datetime=""> <em> <i> <q cite=""> <s> <strike> <strong> We read every comment and will make every effort to approve each new comment within one working day. To ensure speedy posting, please keep your comments relevant to the topic of discussion, free of inappropriate language and in-line with the editorial integrity of this newsroom. If not, your comments may not be published. Thanks for commenting!