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.
Sarepta’s 120 week stability data on both the six minute walk test, as well as, pulmonary function is good news for the Duchenne community. For the 12 young men in the study, we are thrilled. And for those waiting, we see hope on the horizon. We congratulate Sarepta for their continued commitment to people with Duchenne and we hope the FDA…Continue
Added by Pat Furlong on February 6, 2014 at 1:30pm — No Comments
An update on developing "Draft Guidance on the Clinical Investigation of drugs and biologics for Duchenne and Becker muscular dystrophy"
One of PPMD’s priorities is speeding development of reliable diagnostics and safe and effective…Continue
Added by Ryan Fischer on January 28, 2014 at 2:00pm — No Comments
Patrick was 8 years old. Steroids were not recommended. There was no imaging and no way to predict his loss of ambulation. Sure, he fell occasionally. Some days more than others. There was one day in the summer of 1988. We had been active most of the day. We went swimming, to the mall, and ended the day with a cookout. Patrick walked and walked. He asked me to help him to the bathroom. He fell. It was one of those FALLS, as if his legs were pulled out from under him. I had no excuses in my…Continue
Added by Pat Furlong on January 23, 2014 at 9:00am — No Comments
Prosensa will organize a patient focused webinar on Tuesday, January 21st at 8 AM ET / 14hrs CET. Practical details required to dial in for this webinar are provided on the Prosensa website at: http://ir.prosensa.eu/events.cfm (open additional information by clicking on it).
If you have any questions that you would like to see answered during the webinar, these can be…Continue
Added by PPMD on January 20, 2014 at 9:31am — No Comments
A family of 11…9 kids (the youngest with Duchenne), 2 parents and ALL of them participated in last weekend’s Walt Disney World Marathon. Bob McDonald, a PPMD Board Member, and each member of his family ran in at least one of the Disney races. Even his youngest son Mark, with Duchenne, participated via wheelchair. Everyone in the family took a little time and effort (okay, maybe a lot!) and did something to help end Duchenne.…
Added by Will Nolan on January 16, 2014 at 10:51am — No Comments
You are invited to join us in Washington (February 23-25) for what is going to be another critical year in Duchenne advocacy. As you know our efforts last year were focused on collecting cosponsors for the MD-CARE Act (MDCA) reauthorization. To date we stand at …Continue
Added by Ryan Fischer on January 16, 2014 at 10:00am — No Comments
No one wants to be sick or go to the Emergency Room unless they absolutely need to. Keeping your lungs clean and your breathing easy will help you to avoid the Emergency Room. Unfortunately, winter is the time for respiratory illnesses and colds. During a respiratory illness/cold, your weak cough can become…Continue
Added by Kathi Kinnett on January 16, 2014 at 9:00am — No Comments
“We will fight for our sons, we will unite for our sons, and yes, we will run for our sons.”
With those words, Mike Gaglianone, our parent speaker at our team pasta dinner, kicked off an incredible weekend at the Walt Disney World Marathon Weekend January…
Added by Nicole Herring on January 15, 2014 at 3:30pm — No Comments
Last week, Pat Furlong and I were invited by the FDA to their meeting on Complex Issues in Developing Drugs and Biological Products for Rare Diseases. I was asked to participate on the Tolerating Risk and Uncertainty in Pediatric Clinical Trials Panel, in recognition of the work PPMD has done…Continue
Added by Holly Peay on January 15, 2014 at 11:00am — No Comments