Every year on the Saturday night of the Connect Conference, PPMD hosts a final dinner. Usually there is a speaker or two, dinner and drinks and a last moment to talk with friends.
Added by Kimberly Galberaith on May 13, 2014 at 1:30pm — No Comments
The process for writing a Draft Guidance on Duchenne for the FDA and industry is in full swing.
As you know, the purpose of this guidance is to assist…Continue
Added by Ryan Fischer on May 9, 2014 at 9:30am — No Comments
Originally signed into law in 2001, the MD-CARE Act has birthed key discoveries in the muscular dystrophies, including the scientific justification for why exon skipping therapies could potentially work in humans.
Without it being…Continue
Added by Ryan Fischer on May 8, 2014 at 4:30pm — No Comments
A recent PPMD-funded study at Cedars Sinai Medical Center in Los Angeles (published in the May 7, 2014, online issue of Neurology®) demonstrates that those with Duchenne have impaired circulation in their muscles, which may contribute to muscle weakness, and that the approved vasodilator tadalafil can restore that…Continue
Added by PPMD on May 7, 2014 at 4:00pm — No Comments
This past year has brought many new and exciting changes to DuchenneConnect, the robust and cutting-edge registry and resource that connects Duchenne and Becker patients with actively recruiting clinical trials and research studies, and educates patients and families about Duchenne and Becker research. The registry, which was established in 2007, has matured beyond a standard registry and into an innovative…Continue
Added by Ann Martin on May 7, 2014 at 11:32am — No Comments
As we finalize the agenda for this year’s Connect Conference, I am once again amazed at how much of a challenge it is to squeeze in all of the talks on different therapeutic approaches to treating Duchenne—in fact, there are too many to cover comprehensively this year. Sometimes it’s good to remember that mutation-specific approaches like exon skipping, although promising and…Continue
Great investor call from Sarepta this morning on the heels of the news about positive signals from the FDA for submitting a new drug application! We are speaking with the company this morning and will release a detailed statement this afternoon. Congratulations to everyone!
Thanks to your efforts, we closed the House and Senate funding letters with the support of 30 House members and 12 Senators. Great work! We appreciate everyone who sent emails and made calls to their members of Congress.
The members below…Continue
Added by Ryan Fischer on April 15, 2014 at 9:51am — No Comments
Unfortunately, the care of people living with Duchenne muscular dystrophy differs from country to country, from state to state, and even from center to center. Email and stories that we get daily confirm that, despite the availability of Care Standards there is still significant practice variability, including centers and locations where clinicians…Continue
PPMD is thrilled to award two exploratory grants as part of our ongoing grant program. Peter Arthur of the University of Western Australia was awarded $50,000 to finish testing a molecule called procysteine in animal models in preparation for a human clinical trial. Procysteine is an antioxidant that has…Continue
Right now, there are more than 10-12,000 young people diagnosed and living in the United States with Duchenne. These young people are being cared for by clinics both near and far. Every one of them deserves the best care and treatment possible. Families want care and treatment to be transparent; they want to know who is delivering care to their child, what comprehensive care looks like, and which centers are delivering that care. For this reason, a group of experts agreed to give families…Continue
As a community, we must continue to demand that the FDA use all available tools given to them by Congress to speed the approval of new therapies to all who so desperately need them. The voices of patients and caregivers are critical to the process of drug development and we believe the message is being heard.
Here are 3 simple actions you can take to support & amplify that message:
Added by Ryan Fischer on March 19, 2014 at 12:10pm — No Comments
For years our community has been making noise so that the scientific and medical communities would listen and focus on Duchenne. They're listening – now we need to talk! Filling out a survey or doing an interview about your family's experiences with Duchenne creates important data. This data…Continue
Added by Holly Peay on March 18, 2014 at 9:30am — No Comments
Added by PPMD on March 18, 2014 at 9:00am — No Comments
Though the MD-CARE Act remains our top priority, we need to ensure that agencies receive adequate funding and direction for the programs that are so critical to our community. One tool we use to ensure that funding and direction is our Congressional funding letter. It has made a tremendous impact on appropriations.
To illustrate this point, let’s look at how our yearly funding letters have impacted the NIH’s…Continue
As a mother who lost two sons to this dreadful disease, I am driven to find every way possible to help bring new therapies to families, to end Duchenne. PPMD supports and agrees with all parents urging the agency to accept Sarepta’s submission for the approval of eteplirsen, as well as any other therapy that shows similar promise. The voices of patients and caregivers are critical to the process of drug development and we believe the message is being heard.
Added by Pat Furlong on March 7, 2014 at 9:30am — No Comments
We all know that the heart is a muscle too! Most people and parents living with Duchenne feel that care for the heart, as defined by the Care Considerations, is not as current or as aggressive as they would like. In 2009, when the Care Considerations were published, cardiac care was very…Continue
Added by Kathi Kinnett on March 5, 2014 at 1:30pm — No Comments
It's been just one week since the 2014 Advocacy Conference, and we have already upped the number of cosponsors for the MD-CARE Act! We are now at 85 in the House & 21 in the Senate. Please take a moment to reach out to our newest cosponsors…Continue
During my years of clinical practice, I was repeatedly asked, “Is there something that I can feed my child to help slow down, or stop, the effects of Duchenne?” The absolute answer is – we don’t know. But a new article out this week has endeavored to address this question.