PPMD and a coalition of Duchenne foundations continue to work together on preparing for the Sarepta Ad Comm. As of today, we are still waiting on a new confirmed date. With the postponement of the Ad Comm, the Congressional letter to FDA remains open. Has your House Members signed on…Continue
Added by Ryan Fischer on January 25, 2016 at 11:30am — No Comments
There has never been a moment like this in our Duchenne community. Today the FDA released briefing documents on the application for eteplirsen. It is clear that we continue to face a serious regulatory challenge as a community.…
In October, PPMD held a FACES Roundtable in Houston that included an incredible line up of global experts in care and research. In an effort to ensure this information reached families beyond Houston, we recorded the sessions. Special thanks to Rachel and James Poysky for pulling together such a wonderful event.
Stay tuned for more roundtable…Continue
Added by Ryan Fischer on December 10, 2015 at 3:30pm — No Comments
Come and advocate for Duchenne directly with members of Congress.
PPMD's Annual Advocacy Conference, held in the first quarter of each year, is the only officially organized gathering on Capitol Hill for the Duchenne community. Families…Continue
Added by Ryan Fischer on December 3, 2015 at 10:54am — No Comments
We are thrilled to report that last night the Ensuring Access to Clinical Trials Act of 2015 (EACT) passed a vote in the House of Representatives (after passing the Senate by unanimous consent in July) and will go to the President for his signature!
This a result of everyone who contacted their Representative – over 400 families in…Continue
Added by Ryan Fischer on September 29, 2015 at 11:08am — No Comments
URGE THE SENATE TO PRIORITIZE CURES!
One minute of your day could mean millions of minutes for those diagnosed with Duchenne.
Today, patient advocates from across the country will unite in a day of action to urge the Senate to make medical innovation a top priority.
Earlier this year the Duchenne…Continue
Added by Ryan Fischer on September 25, 2015 at 9:30am — No Comments
As we make advancements in Duchenne drug development, the potential therapeutic opportunities for Duchenne patients across the spectrum of the disease continues to grow — as do the opportunities to participate in clinical trials. This is a hopeful and exciting time for our Duchenne community and we must ensure unnecessary barriers don’t hamper clinical…Continue
Added by Ryan Fischer on September 18, 2015 at 10:29am — No Comments
Ad Comm. It’s a term our community will be hearing a lot this fall as companies get closer to the possible approval of treatments for Duchenne. But what is an Ad Comm and how does it involve and affect you or your child?
Added by Ryan Fischer on August 11, 2015 at 12:00pm — No Comments
People around the world will help participate in the second annual World Duchenne Awareness Day on September 7th. There are a variety of ways you can get involved with this international effort – how will you raise awareness in your community?…Continue
Added by Ryan Fischer on August 7, 2015 at 10:30am — No Comments
PPMD has been honored to lead the Duchenne community in developing patient-focused tools like our draft guidance and our benefit/risk surveys to help accelerate the FDA review of Duchenne treatments. Just last week, FDA issued its landmark draft guidance on Duchene therapy development, a document heavily informed by the project submitted nearly one year ago and led by…Continue
Added by Ryan Fischer on June 19, 2015 at 1:00pm — No Comments
BioMarin has provided the following update on redosing of drisapersen for a new clinical trial. Ambulation will not be an inclusion or exclusion criterion for enrollment, but boys will need to have been previously dosed as part of a clinical trial.
BioMarin Update on Duchenne Muscular Dystrophy…
Added by Ryan Fischer on April 14, 2015 at 9:00am — No Comments
Earlier this month Sarepta announced a change in leadership, with CMO Ed Kaye taking over as interim CEO.
Following that announcement the team at Sarepta organized a call with Duchenne patient organizations and foundations to update them…
Added by Ryan Fischer on April 13, 2015 at 11:00am — No Comments
Have your members of Congress signed onto the Duchenne funding letter yet?
Please review the list of Senate and House members who have signed onto the Duchenne funding letter being circulated in both chambers.
Our incredible community is fighting the war on Duchenne on many fronts. These fronts include funding early science and breakthrough translational research, demanding optimal care for all those diagnosed with Duchenne –and changing public policy. Much of those gains on the battlefield have happened due to our intensive focus on advocacy and passage of the…Continue
Added by Ryan Fischer on February 19, 2015 at 10:00am — No Comments
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! Just look at the ‘to-do’ list:
Added by Ryan Fischer on January 28, 2015 at 1:30pm — No Comments
A high school student (Caleb SIzemore) is asking for help with a project about inclusion.
Caleb is doing a senior thesis on avenues to increase social support for boys with Duchenne, based on his experience living with Duchenne and on research. He needs more responses to finalize the project.
Sharing this survey from our friends at UPPMD. Please take a moment to fill it out. It is extremely short.
News: short online questionnaire on Duchenne and epilepsy.
Dear parents, boys and young men,
We want to ask your attention for a scientific research project on Duchenne muscular dystrophy and epilepsy.…Continue
Added by Ryan Fischer on December 18, 2014 at 9:52am — No Comments
Parent Project Muscular Dystrophy (PPMD) is urging Congress to build upon gains in recent years to further strengthen and enhance programs to ensure the patient voice is heeded during the drug development process during a Congressional briefing tomorrow, Tuesday, December 9th at…Continue
Added by Ryan Fischer on December 8, 2014 at 1:30pm — No Comments
PPMD and other leaders in the rare disease community were asked by PhRMA to participate in a blog conversation about how to advance the science of…
Added by Ryan Fischer on November 20, 2014 at 11:30am — No Comments
Dr. Janet Woodcock, Director of FDA's Center for Drug Evaluation and Research (CDER) presented the opening keynote at NORD's Rare Diseases and Orphan Products Breakthrough Summit in Washington, DC on October 21.
Her remarks focused on how the patient advocacy community can most effectively accelerate therapy development along the drug development…
Added by Ryan Fischer on October 23, 2014 at 9:30am — No Comments