Action Alert: Tell the Senate to take action on MD-CARE Act!
Congress has returned from their Summer recess and it is time to re-engage them on the MD-CARE Act! We need each member of our community to reach out to your Senators and urge them to tell leadership to bring the MD-CARE Act to the floor for a vote. Take action now!
PPMD Welcomes Annie Kennedy as Senior Vice President of Legislation & Public Policy
We are thrilled to welcome Annie Kennedy, our new Senior Vice President of Legislation and Public Policy! Annie joins PPMD after being with the Muscular Dystrophy Association (MDA) for 25 years, first as a camp volunteer and as an employee for the last 16 years. Annie’s focus will be on advancing public policy, addressing transition issues for people with Duchenne, and working on newborn screening issues.
PPMD Submits Cardiac Recommendations to FDA
During discussions at a working group meeting designed to explore contemporary issues in Duchenne cardiology, the FDA indicated interest in receiving recommendations for cardiac surveillance and cardiac markers, to be included in current and future Duchenne clinical trials. In response to those questions, recommendations were developed by the Expert Working Group and submitted to the FDA for consideration for inclusion in the draft guidance.
End Duchenne eNews: The YOU in Teamwork
Catch up on the latest research and community updates in this month's End Duchenne eNews, and learn how you can help us tackle Duchenne!
The Breath of Life: New 3-Part Pulmonary Webinar Series
The lungs require the strength of many muscles to help a person to breathe and cough. As people with Duchenne grow older, the respiratory muscles, like the skeletal muscles, become weaker. Like the heart, every person with Duchenne will have pulmonary issues of some type. This is such an important area of care and concern that we pulled together pulmonary experts from across the US to address important issues during a webinar series this fall.
Welcoming Drs. Dongsheng Duan & Jill Rafael-Fortney to PPMD's Scientific Advisory Committee
We are excited to welcome Dongsheng Duan, PhD and Jill Rafael-Fortney, PhD to our Scientific Advisory Committee. The committee, chaired by PPMD Senior Scientific Advisor Dr. Lee Sweeney, expertly reviews every grant that comes in and offers funding or advice on how to proceed to garner funding in the future.
Cincinnati Children’s Named Certified Duchenne Care Center By Parent Project Muscular Dystrophy
Congratulations to Cincinnati Children's Hospital Medical Center on becoming a Certified Duchenne Care Center! Dr. Brenda Wong and team have touched the lives of so many families in this community and are a leader in comprehensive care. Thank you for your hard work!
BREAKING NEWS: Houses Passes MD-CARE Act Amendments!
The House of Representatives today approved the landmark MD-CARE Act so the law can continue getting results for all patients impacted by Duchenne. We will be urging the Senate to promptly take up and pass this bill so this much-needed update to the law can be made as quickly as possible.
Duchenne Cardiac Working Group Recap
Care of the heart in Duchenne is not a simple business. Fortunately, more than 30 representatives from the cardiac care, cardiac research, the NHLBI and the FDA agreed, and were gracious enough to give two days of their very busy lives to discuss just these issues in a recent meeting convened by PPMD.
PPMD's Pat Furlong on BioCentury This Week
Thank you "BioCentury This Week" for having PPMD Founding President & CEO Pat Furlong on its 200th episode this weekend! You can watch Pat's segment online, where she shares her personal story in founding PPMD and why we will not rest until every person has a treatment to end Duchenne.
No Exon Left Behind
PPMD has a decade-long history of supporting exon-skipping, but what exactly is it? How can we bring the technology to less common deletions and duplications as quickly as possible?
PPMD appreciates FDA’s interest in these efforts to provide the agency with stakeholder views about trial processes.
August 18, 2014 | Read more