WASHINGTON, Feb. 24, 2014 /PRNewswire-USNewswire/ -- More than 100 advocates seeking to end Duchenne muscular dystrophy (Duchenne) are converging on Washington for the annual Parent Project Muscular Dystrophy (PPMD) Advocacy Conference to urge Congressional action on key legislation and to help accelerate the Food and Drug Administration's (FDA) review of candidate Duchenne therapies.
The annual PPMD Advocacy Conference – a February Washington mainstay as recognizable as shadow-seeking groundhogs and arrow-shooting cherubs – has been a key component of the organization's success leading the way to achieve meaningful results and progress to end Duchenne. This year's Conference is also timed with Rare Disease Week, which continues to grow as an opportunity for the rare disease community to share their stories.
"Thanks to the tireless commitment of our advocates and a comprehensive, focused, and results-oriented policy agenda, we are making progress in achieving our singular goal of ending Duchenne," said PPMD Founding President Pat Furlong.
"By focusing on achieving clear results and progress, our advocates are moving the needle both with elected representatives in Congress as well as with agency officials at the FDA so effective and accessible therapies for Duchenne can be delivered to patients in need as quickly as possible," Furlong added. "We learned many years ago that it takes an aggressive and thoughtful strategy to move the needle in research, care, and access."
In addition to meetings with Senators, Representatives, and staff on Capitol Hill to urge them to update the landmark Muscular Dystrophy Community Assistance, Research, and Education (MD-CARE) Act law that has helped transform research and clinical care, this year's conference will feature a half-day session focused on developing clear guidance to inform Duchenne clinical trials to make for the fastest possible review of hoped for Duchenne drug applications by the FDA.
Such guidance, made possible under the FDA Safety and Innovation Act (FDASIA), provides direction to drug developers as to milestones that must be achieved in clinical studies. By providing such direction before a trial begins, industry can better design and orient trials, saving time and resources.
"PPMD actively supported many of the patient-focused drug development provisions included in FDASIA. Now, we are using these new powers bestowed upon the FDA to develop community-informed guidance that we will submit later this spring to the FDA for review," Furlong said.
The conference also includes a special briefing convened by PPMD and allies the Muscular Dystrophy Association (MDA) and the Foundation to Eradicate Duchenne (FED) focused on the immeasurable impact the MD-CARE Act has had in lengthening and improving quality of life and generating research breakthroughs. PPMD is leading the effort to update the law so it remains focused on areas of greatest need.
"The MD-CARE Act has been the engine behind much of our success over the past 12 years. Like many laws, it requires regular updating, and we urge Congress to complete this process this year so the act can continue driving innovation and improving quality of life for all persons impacted by muscular dystrophy," Furlong said.
About Parent Project Muscular Dystrophy
Duchenne is a fatal genetic disorder that slowly robs young men 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 young men 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 boys with Duchenne live longer, stronger lives. We will not rest until every young man 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.
SOURCE Parent Project Muscular Dystrophy