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An update on developing "Draft Guidance on the Clinical Investigation of drugs and biologics for Duchenne and Becker muscular dystrophy"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
We are heartbroken to learn more discouraging news about another Duchenne therapy today, HT-100 from Halo Therapeutics. This community continues to face tremendous disappointment.
As part of Halo’s efforts to accelerate HT-100 development, Halo was conducting a toxicology study in dogs in parallel with the clinical program. In this study, the dogs got higher exposures (levels of…Continue
Analyses and assessment of next steps are ongoing
PPMD just received the following update from GSK regarding the current status of their drisapersen study:
Dear Patient Group Representative,
On the 20th of September this year, we shared the results about our Phase III study (DMD114044) of drisapersen in boys with Duchenne Muscular Dystrophy – we did not see a statistically significant difference in 6 Minute…
Added by PPMD on December 19, 2013 at 10:30am — No Comments
Each month for the last couple of years we have featured people with Duchenne who have made an impact on their community and our community in some significant way. Whether working with their parents to host a fundraising event, participating in a race, speaking in Washington, D.C., or sharing their story…Continue
Added by Will Nolan on December 16, 2013 at 2:30pm — No Comments
At yesterday’s Duchenne Policy Forum, Vice President of Research Sharon Hesterlee, PhD, tweeted throughout the day, reporting to the community what transpired during this historic meeting. We have compiled these tweets and fit them into the…Continue
12/12/13 8:30 AM
There are days when the stars align just right. Seems to me, December 12, 2013 was one of those days.
Eighteen members of the FDA arrived and were seated around a U-shaped table near the front of the…Continue
Why the December FDA Policy Forum is important to every Duchenne family – from rare mutations to Exon 51
Guest post Lance Hester. Lance is a PPMD Board Member and lives in Gig Harbor, Washington, with his wife, Janelle, and their two boys, Brayden and Micah. Micah was diagnosed with Duchenne in 2005.…Continue
Added by PPMD on December 9, 2013 at 1:00pm — No Comments