Presentation October 11th 2016

Current and future treatment and benefits of early diagnostics


Duchenne Parent Project

Duchenne Muscular Dystrophy is a progressive and ultimately fatal genetic disorder affecting 1 in every 3500 newborn boys. The organisation Duchenne Parent Project was set up by Duchenne parents who decided to shoulder responsibility and become an active partner in the development of viable treatments or a cure for this disease. They became funders, advisors and advocates. Duchenne Parent Project NL funds research project around the globe and has funded around 100 research projects in Academia as well as in Industry. Duchenne Parent Project has played a role in raising awareness, collecting data on patient and caregiver preferences, the development of outcome measures, standards of care and bridging the so-called ‘Valley of Death’ in drugdevelopment. Duchenne Parent Project is also involved in the development of exoskeletons and other technologies for DMD and plays an important role in improving care, setting up standards of care and the dissemination of this information.

* http://www.enmc.org/files/9613/7568/4843/ENMC_workshop_195_NBS_report_NMD_Jul_2013.pdf



The presentations are available for educational exchange purposes only. Products mentioned in the presentation may not be available in your country. Please consult your local PerkinElmer representative for the products available in your country