CHICAGO, June 30, 2017 /PRNewswire-USNewswire/ -- Parent Project Muscular Dystrophy (PPMD), a nonprofit organization leading the fight to end Duchenne muscular dystrophy (Duchenne), will host their annual Duchenne Drug Development Roundtable during the organization's 23rd Annual Connect Conference being held in Chicago, Illinois.
Duchenne muscular dystrophy is the most common fatal genetic disorder diagnosed in childhood, affecting approximately one in every 5,000 boys.
Established more than seven years ago, PPMD's Duchenne Drug Development Roundtable (DDDR) is a group of committed innovators representing industry and relevant stakeholders that has the goal of accelerating the development of meaningful treatments for Duchenne through open discussion and working collaboratively in the precompetitive space to minimize duplication.
This year's annual DDDR has never been more important or timely. There are now more than three dozen publicly and privately traded companies in Duchenne; more than a dozen therapies in late stage clinical trials; and a robust pipeline representing an array of therapeutic approaches not far behind. The Duchenne community also has crossed into the post-approval space with the accelerated approval of EXONDYS 51 in September of 2016 and the recent approval of EMFLAZA this past spring.
But, according to PPMD Founding President and CEO, Pat Furlong, "The last two years – two years of intense product reviews for our Duchenne therapies – have provided us with new challenges and opportunities. These reviews – coupled with PPMD's policy engagement around the Duchenne community-led Draft Guidance, key provisions within 21st Century Cures, Benefit-Risk studies, and PDUFA VI discussions – have resulted in an opportunity to take what has been learned from all trial experiences and work together to become even more focused and efficient. PPMD and the leadership of our DDDR felt that this is a strategic inflection point for Duchenne; a moment to bring industry partners together to assess how these 'regulatory learnings' and new policies might best be applied back into our pipeline and into clinical trial design."
To achieve this, the DDDR Steering Committee mapped out a series of three one-day meetings that were held earlier this year. Each included leaders and innovators from outside the Duchenne space to present on opportunities to enhance our existing trial designs, tools, and infrastructure.
The meeting series topics include:
- Clinical Trial Readiness – Patients, Families, and Treating Physicians
- Optimizing Clinical Trial Design
- Clinical Trial Readiness – Sites & Investigators
Furlong believes that the DDDR meeting at PPMD's Connect Conference is a unique moment in the Duchenne calendar each year, providing companies and stakeholders a chance to truly connect. Says Furlong, "We appreciate each company that has come to the table to lean in even further and we look forward to the opportunity to work together as a community to explore the innovations and opportunities that are raised through ongoing discussion and exploration. It is our hope that the DDDR will continue to assist PPMD and the Duchenne community, working together to drive forward collaborations and projects that will benefit all partners working in this space and yield effective therapies that are accessible to all who need them."
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. PPMD believes strongly in the power of connecting. The 2017 Connect Conference is sponsored, in part, by Elite Sponsors PTC Therapeutics, Santhera Pharmaceuticals, and Sarepta Therapeutics.
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.
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SOURCE Parent Project Muscular Dystrophy