Upcoming Webinar: Efficacy of Catena®/Raxone® (Idebenone) on Respiratory Outcome in Duchenne
Santhera will present results from its Phase III study with Catena®/ Raxone® (Idebenone) in Duchenne during a webinar hosted by PPMD on Wednesday, October 15th at 12pm eastern.
President Signs MD-CARE Act Amendments Into Law
Thank you again to everyone in this community who helped advocate for the passage of this critical legislation! We couldn't be more proud to be on your team!
The MD-CARE Act: Reflections & Projections
Sometimes it is worth taking a moment to reflect so that as we continue to plan ahead we can learn from not only our mistakes, but our victories. Our advocacy initiatives continue to grow and we won’t slow down. We can’t. And we hope you’ll join us as we continue to use our voice in D.C.
Duchenne Newborn Screening
Our Duchenne community has therapies in today’s pipeline that will be most effective when administered early. Our formalized efforts to move towards Duchenne newborn screening began several years ago; our community push begins now.
Upcoming Webinar: Prosensa Update about Drisapersen & Other Programs
UPPMD will host a webinar with Prosensa on Thursday, October 2 at 12 pm EDT.
During the webinar, Prosensa will answer questions from the Duchenne Community and give an update.
BREAKING NEWS: MD-CARE Act Passed!
Today – September 18, 2014, our Duchenne community once again made history by demonstrating that nothing can stop us. Today, the MD-CARE Act Amendments of 2014 passed! WE DID IT!!
PPMD Names Seattle Children's Hospital Certified Duchenne Care Center
Seattle Children's is the fourth center to be certified by PPMD, recognizing the hospital's dedication to improving care for people living with Duchenne.
End Duchenne eNews: Here to Help
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!
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.
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.
Today September 18, 2014, our Duchenne community once again made history by demonstrating that nothing can stop us.
September 18, 2014 | Read more
October 15, 2014