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!
MD-CARE Act: We are not done yet! Tell the Senate to act this September.
Sure, we’re in the lazy days of summer and Congress is on recess, but now is not the time to slow down on the MD-CARE Act. Let’s keep our momentum going. Take a minute to thank the House for passing the bill and tell the Senate why we need them to take up the legislation this September to do the same!
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 three-part 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?
One Step Closer
Progress! Today the House Energy & Commerce committee voted unanimously to advance H.R. 594, the MD-CARE Act Amendments of 2014. This action moves the bill to consideration and hopefully passage by the full House of Representatives.
PPMD President Testifies Before Key Congressional Committee
Pat Furlong, Founding President and CEO of Parent Project Muscular Dystrophy will testify before the House Energy and Commerce Committee’s Subcommittee on Health as part of the committee’s ongoing 21st Century Cures Initiative on July 11 at 9am eastern. She is one of four patient advocacy representatives selected to testify in a hearing entitled “21st Century Cures: Incorporating the Patient Perspective."
Outcomes Meeting: Laying a Foundation for Outcome Measures
At PPMD's pre-conference meeting, a group of therapists and industry representatives met to discuss the importance of standardizing outcome measures.
The best part of the Connect Conference is being there, being together, connecting with the Duchenne community, the annual reunion. HOPE and CHANGE filled the air.
July 2, 2014 | Read more