PPMD & FED Award $1 Million Grant to ReveraGen BioPharma
Parent Project Muscular Dystrophy (PPMD) is excited to join forces with Foundation to Eradicate Duchenne (FED) to award ReveraGen BioPharma a $1 million grant to help fund the chronic toxicology program for VBP15, a combined action drug that addresses multiple levels of Duchenne.
Upcoming Webinar: Dystrophin 101
Join us for a webinar on "Dystrophin 101: Everything You Always Wanted to Know About the Duchenne Protein (And Were Not Afraid to Ask)" on March 3, 2015 from 1pm to 2pm ET.
Take Action While Advocates Are Storming the Hill!
This week is Rare Disease Week and over 150 Duchenne Advocates are in Washington, D.C. spreading the message that Duchenne matters and must remain a priority to the government. Please take a minute to re-emphasize our message. Your short email will strengthen the work of your fellow Duchenne advocates storming the Hill today.
Advocacy Matters: Getting Results to End Duchenne
The recording of our "Advocacy Matters" webinar is now available. Watch now for an overview of PPMD's advocacy history, our advocacy conference "ask" this year, and what you can expect over the next few days as we kick off our 2015 Advocacy Conference in Washington, DC.
End Duchenne eNews: Raise Your Voice
Catch up on the latest research and community updates in this month's End Duchenne eNews!
PPMD Awards University of Missouri $50,000 Exploratory Grant to Explore SERCA2A in Duchenne Dog Study
PPMD has awarded a $50,000 Exploratory Grant to University of Missouri to test SERCA2A gene therapy as a potential treatment for dogs with Duchenne. Dr. Dongsheng Duan, a longtime researcher in Duchenne and a member of PPMD’s Scientific Advisory Committee, leads the project.
Your Action Plan for the Muscular Dystrophies
The 2015 Action Plan from the Muscular Dystrophy Coordinating Committee (MDCC) is now available for public comment. PPMD encourages all members of the Duchenne community to provide comments, in particular to ensure that the patient and family voice is heard in the Action Plan.
PPMD Names University of Iowa Children’s Hospital a Certified Duchenne Care Center
With this certification, PPMD recognizes the quality of the neuromuscular care for children living with Duchenne provided by the multidisciplinary team led by Katherine Mathews, MD. This team illustrates the dedication to the Duchenne community that the certification program seeks to promote.
PPMD Awards UCLA $50,000 Exploratory Grant for Evaluation of Exon Skipping Enhancers in Duchenne
PPMD is excited to award Dr. Carrie Miceli and her team at UCLA’s David Geffen School of Medicine and College of Letters and Science, a $50,000 exploratory grant to continue their evaluation of exon skipping boosters for the treatment of Duchenne.
Get Active in Advocacy
2014 was a historic year in our community. 2015 promises to be another critical year in Duchenne and we need you to help keep the momentum going! With such a big year, it is more important than ever to get involved. Our Annual Advocacy Conference is one month away. We urge everyone to lend your voice, your story, to our efforts in Washington. You don’t have to travel to Washington to be an effective advocate.
FDA Acknowledges the Importance of Benefit-Risk Preferences: Why this Matters to Duchenne
Recently the FDA approved a device used to treat obesity, based in part on the results of the preferences study that showed that patients would accept uncertainty about benefits. An FDA spokeswoman said that the FDA believes patients and caregivers who live with a condition should have a say when approving a product, and may have a different view about striking the right risk-benefit balance. This could have incredible significance in the Duchenne community and makes our ongoing benefit/risk work all the more critical.
Important Vaccinations Recommendations
Vaccinations are important to protect us against infection and disease but they are also confusing. Adding Duchenne medications, pulmonary, and cardiac function to the picture adds even more confusion. In order to answer the regularly asked questions, “to give or not to give, when to give, how to give and what to give,” we have teamed up with the CDMD at UCLA to provide recommendations.
BMS & The Voice of the Patient
It is a good day when a global biopharmaceutical company commits to focusing on Duchenne. We are grateful that Bristol-Myers Squibb (BMS) has added another possibility for our toolbox of hope!
Meet Our 2015 PPMD Adult Advisory Council
The PPMD Adult Advisory Council (PAAC) serves to ensure that the voices of adults living with Duchenne and Becker muscular dystrophy are reflected in PPMD’s legislative, policy, and program activities. PAAC members serve in a formal advisory capacity for their respective term and maintain leadership over a PAAC initiative throughout that term; PAAC initiatives are determined by the PAAC membership and PPMD Executive Team.