Finding gratitude in the bittersweet
"I look at this list of what we have done as a community, and my heart swells with gratitude for all of you that have stood alongside us — some for 20 years, some maybe just for the last few months. Our approaches may differ, but our goal is the same: end Duchenne.
Despite all the heartaches that we experience and will continue to experience, we see strength…together. For that I am always grateful."
PPMD and Santhera Pharmaceuticals Team Up on New Benefit/Risk Study Focused on Pulmonary Therapies
We are proud to announce a collaboration with Santhera Pharmaceuticals on a new benefit/risk study that will focus specifically on patient and caregiver preferences regarding pulmonary therapies in the disease, and will be based on data from Santhera’s successful phase III clinical trial of idebenone.
21st Century Cures: A sign that the tide could be turning
Would you be surprised if we were to tell you that it takes an average of 10 years and one billion dollars to yield a single new therapy, and that many of our nation’s leaders admit that our scientific and clinical institutions and the agencies that regulate them are set up in an antiquated and inefficient way? Probably not, yet none of us in the #Duchenne community has that luxury of time or money.
But there is a sign that the tide could be turning.
Help us bring the ABLE Act to the Floor!
The Achieving a Better Life Experience (ABLE) Act (S. 313/H.R. 647) has been 8 years in the making and has the support of more than 85% of Congress; please help us get it over the finish line! We need you to make 4 easy phone calls, tweets, and posts to ensure ABLE gets scheduled for a vote!
PPMD Names Baltimore’s Kennedy Krieger Institute a Certified Duchenne Care Center
Kennedy Krieger Institute in Baltimore, Maryland is the fifth center to be certified by PPMD, recognizing the Institute’s dedication to improving care for people living with Duchenne.
End Duchenne eNews: Patients Are Waiting
Catch up on the latest research and community updates in this month's End Duchenne eNews!
Webinar Recording & Summary: Duchenne Pulmonary Care for Tweens and Teens
A summary and recording of last week's pulmonary care webinar is now available. Thank you to Dr. Jonathan Finder (Children’s Hospital of Pittsburgh) and Dr. Oren Kupfer (Children’s Hospital Colorado) for such an informative hour.
Patients Are Waiting...
Thanks to the dozens of you who participated in PPMD’s benefit/risk project and took the time to share your story, we have been able to compile your responses and will be providing the FDA with “Patients Are Waiting…” These are your stories, your messages to the Agency demanding that they use all the tools at their disposal to make decisions efficiently. We don’t have time.
PPMD Submits Letter to FDA Regarding Latest Sarepta Update
Today’s press release and investor call from Sarepta have sent a ripple of angst and sadness through our community. Moments ago, PPMD sent a letter to the FDA that reflects our organization’s belief that safety and rigor have been demonstrated throughout this process. Further, we believe that any strengthening of data packages that causes delays (as is now being requested by the FDA, as articulated in today’s press release and call) should ensure a streamlined, more expeditious approval process for follow-on exons.
PPMD-Funded Gene Therapy Study Improves Walking Ability in Muscular Dystrophy
Dr. Jerry Mendell of Nationwide Children’s Research Institute recently presented data from a ground-breaking PPMD-funded study demonstrating that a modified virus carrying the gene for follistatin can improve performance on the 6 minute walk test in study participants with Becker muscular dystrophy. This is the first time a gene therapy has demonstrated an improvement in function in a muscular dystrophy.
Webinar Recording: Efficacy of Catena®/Raxone® (Idebenone) on Respiratory Outcome in Duchenne
The recording of our webinar with Santhera Pharmaceuticals is now available. Prof. Gunnar Buyse (University Hospitals Leuven, Belgium; PI of the study) and Thomas Meier PhD (CEO of Santhera Pharmaceuticals) presented data of the Phase III DELOS trial of Catena®/Raxone® (Idebenone) in Duchenne.