End Duchenne Day to be celebrated on June 26, 2010: PPMD

Conference Includes Launch of First-Ever Duchenne Therapeutic Development Meeting

Patricia A. Furlong, Founding President and CEO of Parent Project Muscular Dystrophy (PPMD), the largest non-profit organization in the United States focused on finding a cure for Duchenne muscular dystrophy (Duchenne), announced that the Mayor of Denver, Colorado, John W. Hickenlooper, has proclaimed June 26, 2010 "End Duchenne Day" in recognition of PPMD's 16th Annual Connect Conference. Denver is playing host to the country's largest Duchenne-specific, international conference, June 24 – 27, 2010.

The Proclamation from Mayor Hickenlooper reads: "The Connect Conference serves as a way to link the resources and needs of the Duchenne muscular dystrophy community to clinical care providers, policymakers, industry professionals, and the medical research fields."

Researchers, scientists, and Duchenne experts from around the world travel to the Connect Conference every year to share with families the latest in muscular dystrophy research and care. Duchenne, the most common form of childhood muscular dystrophy, is a progressive and fatal muscle disorder affecting boys and young men that causes the loss of muscle function, wheelchair dependency, and a decline in respiratory and cardiac function.  

This year's Annual Connect Conference, though, boasts a new addition to its already robust agenda, launching the first-ever Duchenne Therapeutic Development Meeting. Sharon Hesterlee, Ph.D., Senior Director of Research and Advocacy for PPMD, explains the purpose of this parallel scientific conference: "As new treatments for Duchenne approach or reach the clinical testing phase, there is a need for a regular venue to present scientific data, exchange ideas, and develop new collaborations. Over the past several years, many investigators have presented information about Duchenne research at our annual, family-focused Connect Conference -- a service that is invaluable to our families. This year we are adding a full-fledged scientific conference emphasizing therapy development for Duchenne which will provide top scientific investigators, regulatory officials, industry representatives, and follow-on funders, a forum to exchange ideas and findings, and which invites collaboration and unity."

The goals of the Duchenne Therapeutic Development Meeting include: reviewing progress in areas of active therapeutic development for Duchenne, including gene therapy, exon-skipping, utrophin upregulation, and many others; highlighting remaining challenges for individual therapeutic strategies; and showcasing novel techniques and therapeutic ideas.

In addition to launching the Duchenne Therapeutic Development Meeting, PPMD will host the Duchenne Registry and Infrastructure Consortium. This meeting hopes to explore the potential for developing a public-private consortium to aggregate current registry data; pool costs for curation; expand participant enrollment; develop specific modules for additional data points and maintain the ability of industry users to control their own data to the extent required for regulatory compliance.  This exploratory meeting is meant to gauge interest in developing a more sustained effort in this direction, or, if a consortium approach is not feasible or advised, to better understand the needs of industry.

As Ms. Furlong and the PPMD staff gear up for this year's Conference, she commented on what the Mayor's Proclamation and the addition of the new scientific meeting means to the Duchenne community: "Denver is a special place. It is home to some of our community's most active families, as well as, The Children's Hospital, a cutting-edge facility that is taking a leading role in the fight to end Duchenne. To be so warmly embraced by the city and the mayor not only starts off our Annual Connect Conference and our new Duchenne Therapeutic Development Meeting with added enthusiasm and a greater sense of purpose, it raises awareness which is invaluable for a rare disease. We are so looking forward to the wealth of information we anticipate will be shared at both of these conferences next week. It is a truly historic time for the Duchenne community."