Recent MDI Funded Research Projects
In 2010, MDI was funding four pieces of work:
Dr. Matthew Wood, University of Oxford
MDI has supported Dr. Wood for a number of years for his work on enhanced systemic delivery of antisense oligonucleotides for exon skipping in Duchenne muscular dystrophy. This work is trying to improve the efficiency of the technology for exon skipping and will feed into ongoing clinical trials.
Dr. Deniz Yilmazer-Hanke, University College Cork
Dr. Yilmazer-Hanke from UCC extended her study which was due to complete at the end of 2010, “Contribution of the Inflammatory Reaction in the Muscle in Duchenne Muscular Dystrophy to Depression: Development of New Treatment Strategies”. The time extension into 2011 will allow additional testing.
A poster was displayed at the Neuroscience Ireland meeting in September 2010 in University College Dublin about Dr. Yilmazer-Hanke’s research and MDI’s support was acknowledged on this poster.
A poster was displayed at the Neuroscience Ireland meeting in September 2010 in University College Dublin about Dr. Yilmazer-Hanke’s research and MDI’s support was acknowledged on this poster.
Prof. Kay Ohlendieck, NUI Maynooth
Prof. Ohlendieck completed his three year study in 2010, “Identification of Novel Biomarkers in Dystrophic Heart and Muscle Fibres Using Comparative Proteomics”. This work is important as measuring biomarkers can show the effect that a treatment is having. Prof. Ohlendieck is collaborating with Prof. Steve Wilton in Australia, utilising biomarkers to show the performance of the exon skipping technique.
Dr. Bryan Lynch, Central Remedial Clinic
Dr. Lynch received funding from MDI between 2009-2010 to employ a Clinical Research Fellow in the CRC. Dr. Niamh ni Shuibhne took up this position and set up a clinical assessment tool using the same structure as the North Star Database in the UK. This documents issues such as steroid use, cardiac and respiratory management, bone health and home adaptations.
There are 65 boys attending the CRC and 10 under Crumlin’s care. Dr. Ni Shuibhne also issued a quality of life assessment questionnaire to families with a child with Duchenne. 39 questionnaires were returned. The assessment is very useful to measure the impact of Duchenne on children and their families and is also a useful tool to develop services. It is interesting that parents tended to score quality of life as lower than the child did.
The CRC has implemented changes over the last year, including improved cardiac assessment and spirometry to assess respiratory function.
There are 65 boys attending the CRC and 10 under Crumlin’s care. Dr. Ni Shuibhne also issued a quality of life assessment questionnaire to families with a child with Duchenne. 39 questionnaires were returned. The assessment is very useful to measure the impact of Duchenne on children and their families and is also a useful tool to develop services. It is interesting that parents tended to score quality of life as lower than the child did.
The CRC has implemented changes over the last year, including improved cardiac assessment and spirometry to assess respiratory function.
