DMD+Materials+and+Methods

Journal Articles:

Eteplirsen in the treatment of Duchenne muscular dystrophy doi: 10.2147/DDDT.S97635 Date Accessed: Jan-March 2017 Purpose: To examine the efficacy of exon treatment, "Eteplirsen" on the masking/skipping of exon in the region of exon 51.

The TREAT-NMD DMD Global Database: Analysis of More than 7,000 Duchenne Muscular Dystrophy Mutations doi: 10.1002/humu.22758 Date Accessed: Jan-March 2017 Purpose: For visual representation of the prevalence of DMD from global perspective and to identify which populations may be eligible for exon skipping.

Emotional impact of genetic trials in progressive paediatric disorders: a dose-ranging exon-skipping trial in Duchenne muscular dystrophy. doi:10.1111/j.1365-2214.2012.01387 Date Accessed: Jan-March 2017 Purpose: This study examined the emotional effects that families may undergo as they explore and attempt gene therapy and other genetics based trials to treat DMD.

//Eteplirsen for the treatment of Duchenne muscular dystrophy.// doi: 10.1002/ana.23982 Date Accessed: Jan-March 2017 Purpose: This study used exon skipping to do a clinical trial, designed to skip exon 51. A double blind, placebo-controlled study was conducted. The participants, ages 7-13 were given randomized weekly IV infusions half with Eteplirsen and the other half a placebo. After 24 weeks, the presence of dystrophin was measured in all subjects with those receiving treatment having a 22% increase and 0% increase in the control group. After 24 weeks the treatment receiving groups continued and the placebo group was added to the treatment program as well. The study seems to indicate that upon continued treatment, the production of dystrophin increases.

Review Articles:

//Current and emerging treatment strategies for Duchenne muscular dystrophy. // doi:https://doi.org/10.2147/NDT.S93873 (Links to an external site.). Date Accessed: Jan-March 2017 Purpose: A synopsis of current treatment options for DMD.

Abstract (back)

Results Discussion Broader Impact Works Cited