CHICAGO, June 28, 2017 /PRNewswire-USNewswire/ -- Parent Project Muscular Dystrophy (PPMD), a nonprofit organization leading the fight to end Duchenne muscular dystrophy (Duchenne), will convene medical experts from around the world for a workshop exploring inflammation and immunity in Duchenne. This two-day workshop precedes PPMD's 23rd Annual Connect Conference being held in Chicago, Illinois and is sponsored, in part, by PTC Therapeutics, Santhera Pharmaceuticals, Sarepta Therapeutics, Catabasis Pharmaceuticals, and Pfizer, Inc./Bamboo Therapeutics.
Duchenne muscular dystrophy is the most common fatal genetic disorder diagnosed in childhood, affecting approximately one in every 5,000 boys.
In January, PPMD organized a Critical Path Innovation Meeting with the FDA focused on "Inflammation and Immune Response in Duchenne." The goal of the meeting was to begin to explore the role that inflammation plays in Duchenne and how it might be measured. Biomarkers of inflammation were also discussed in terms of differences resulting from age and stage of disease, variability of biopsied muscle site, use of less invasive techniques and tissue for biomarker analyses, the need for robust validation of novel biomarkers, and challenges of differentiating steroid myopathy from underlying disease.
Also discussed was the concept that there may not be a single treatment or dose that is consistently needed throughout the stages of disease or even within an individual with Duchenne. It may be important to tailor therapeutic options. What is needed is a clearer understanding of the inflammation related pathways at a molecular level so that specific inflammatory processes can be targeted at the right time.
This 90-minute meeting helped identify the need for PPMD to convene a more involved workshop to further discuss what is known and unknown, about the role of inflammation and the immune response in Duchenne.
According to Abby Bronson, PPMD's Senior Vice President of Research Strategy, "The agenda for this upcoming meeting will include Duchenne experts, as well as leaders from other disease areas bringing their expertise and experience to the Duchenne community. We look forward to better understanding the role that inflammation and immunity play in Duchenne, and we are anxious to continue discussing potential therapeutic options that will help every single person living with this disease."
PPMD's Annual Connect Conference is the largest, most comprehensive annual, international conference focused entirely on Duchenne. Each year nearly 500 families from around the world gather at PPMD's Connect Conference to learn the latest progress in the fight to end Duchenne. It is also an opportunity for families to gather for support, strength, and camaraderie.
About Parent Project Muscular Dystrophy
Duchenne is a fatal genetic disorder that slowly robs people of their muscle strength. Parent Project Muscular Dystrophy (PPMD) is the largest most comprehensive nonprofit organization in the United States focused on finding a cure for Duchenne muscular dystrophy—our mission is to end Duchenne.
We invest deeply in treatments for this generation of people affected by Duchenne and in research that will benefit future generations. We advocate in Washington, DC, and have secured hundreds of millions of dollars in funding. We demand optimal care, and we strengthen, unite and educate the global Duchenne community.
Everything we do—and everything we have done since our founding in 1994—helps people with Duchenne live longer, stronger lives. We will not rest until every person has a treatment to end Duchenne. Go to www.ParentProjectMD.org for more information or to learn how you can support our efforts and help families affected by Duchenne. Follow PPMD on Facebook, Twitter, and YouTube.
SOURCE Parent Project Muscular Dystrophy