Action Duchenne

Background:Recent short-term clinical trials in patients with Duchenne Muscular Dystrophy (DMD) have indicated greater disease variability in terms of progression than expected. In addition, as average life-expectancy increases, reliable data is required on clinical progression in the older DMD population.Objective:To determine the effects of corticosteroids on major clinical outcomes of DMD in a large multinational cohort of genetically confirmed DMD patients.Methods:In this cross-sectional study we analysed clinical data from 5345 genetically confirmed DMD patients from 31 countries held within the TREAT-NMD global DMD database. For analysis patients were categorised by corticosteroid background and further stratified by age.Results:Loss of ambulation in non-steroid treated patients was 10 years and in corticosteroid treated patients 13 years old (p = 0.0001). Corticosteroid treated patients were less likely to need scoliosis surgery (p < 0.001) or ventilatory support (p < 0.001) and there was a mild cardioprotective effect of corticosteroids in the patient population aged 20 years and older (p = 0.0035). Patients with a single deletion of exon 45 showed an increased survival in contrast to other single exon deletions.Conclusions:This study provides data on clinical outcomes of DMD across many healthcare settings and including a sizeable cohort of older patients. Our data confirm the benefits of corticosteroid treatment on ambulation, need for scoliosis surgery, ventilation and, to a lesser extent, cardiomyopathy. This study underlines the importance of data collection via patient registries and the critical role of multi-centre collaboration in the rare disease field.

Helping children to visualise and understand how sound travels. By Tara Hart, an advanced skills teacher at St John the Divine School, Kennington, London.

Five to SevenVol. 1, No. 5 Science Year 2Grouping materialsTara Hart DykeTara Hart DykeSearch for more papers by this authorTara Hart DykePublished Online:27 Sep 2013https://doi.org/10.12968/ftse.2001.1.5.16908AboutSectionsPDF/EPUB ToolsAdd to favoritesDownload CitationsTrack CitationsPermissions ShareShare onFacebookTwitterLinked InEmail AbstractGROUPING materials is a topic which is rich with opportunities for hands-on experience for children. They are required to use all of their senses in the exploration of common materials around them. FiguresReferencesRelatedDetails 1 September 2001Volume 1Issue 5ISSN (print): 1473-4044 Metrics History Published online 27 September 2013 Published in print 1 September 2001 Information© MA Education LtdPDF download

Regularly reviewing number bonds to 10 and 20 throughout key stage 1 will help children become confident when using and applying them. Rapid recall of these number facts to 10 is a very useful tool when tackling more difficult problems.