by: Parent Project Muscular Dystrophy
This time of year, we are all looking for the perfect gift for our loved ones, but one size doesn’t fit all. This reminds me of Duchenne, where one size doesn’t fit all either. While we have a basic idea of the projected path of Duchenne, we don’t fully understand yet what makes each individual’s journey different.
We need to further unlock our community’s data to unleash clearer answers. That is why PPMD is investing $400,000 into a Duchenne Protein Mapping Project as part of our Biomarker Initiative in 2021. Support this project today and your gift will be doubled, thanks to community partners Team Joseph, Little Hercules Foundation, Ryan’s Quest, and Another Day for Gray who are matching all donations this holiday season up to $200,000.
When we talk about protein mapping, we are talking about better characterization of Duchenne by understanding protein biomarkers. Protein biomarkers may sound a little technical, but let me explain.
Biomarkers are characteristics of the body that can be measured. Some are measured by samples taken from a patient, such as their tissue, blood, urine, or saliva, while others are measured with devices. Your blood pressure is a biomarker. Your oxygen level measured by an oximeter is a biomarker. Imaging can also be used as a tool to measure biomarkers; a tool many families have utilized along their Duchenne journeys. For instance, cardiac MRIs look for biomarkers of heart disease. MRIs can also measure changes in body composition like fat fraction in muscle, a predictor of disease progression and loss of function.
PPMD has funded work in each of these areas to broaden our knowledge base and help us form a better understanding of Duchenne progression and the map that defines it. Your donation today propels us forward in finding clearer answers, faster. Give today and your gift will be doubled.
In 2021, PPMD will support analysis of blood and urine samples collected over the years from ImagingDMD and Leiden University. Samples from well characterized patients who have been part of these natural history studies will be analyzed on SomaLogic’s protein discovery platform. By examining blood and urine on this platform, a tool that can screen for thousands of proteins at once, we aim to find a Duchenne-specific map.
Biomarkers are critical data tools because they can tell us how the body is doing, they can measure the effects of investigational drugs on people in clinical trials and provide insight into why some people progress more rapidly than others. This data could greatly increase our understanding around each individual’s progression in Duchenne and potentially predict and monitor their responsiveness to therapies to help transform the landscape of clinical trials and unleash the breakthroughs of tomorrow.
For so long, our PPMD community has given us their blood, sweat, and tears. By funding this project we make full use of that commitment, and are hopeful that this precious data will yield answers that will influence next steps in research for all ages and stages of Duchenne.
Double your donation today and help us unlock critical data and unleash the possibilities ahead.
Thank you for being part of our PPMD family. We hope you will consider making a gift to support this important work. Unlocking our data together has the ability to unleash new information and propel Duchenne research into a new era.
Warmly,
Eric Camino, PhD
VP, Research and Clinical Innovation
