In light of PPMD’s Cardiac Webinar Series, our Director of Care, Kathi Kinnett answers questions/concerns about Ventricular Assist Devices (VAD).
Added by Kathi Kinnett on October 11, 2012 at 2:30pm — No Comments
These are the words that best describe PPMD’s research plan.
Everything we do goes through this filter to make sure that we are achieving one of these…Continue
Parent Project Muscular Dystrophy recently hosted the first part of our Cardiac Webinar Seriers with Dr. Linda Cripe (Nationwide Children’s Hospital, Columbus, OH) and Dr. Kan Hor (Cincinnati Children's Hosptial, Cincinnati, OH) to discuss monitoring heart disease in Duchenne and carrier moms/daughters.
Added by Kathi Kinnett on October 9, 2012 at 3:30pm — No Comments
With the recent news of Sarepta's promising 48-week data, many have asked how accelerated approval may play into the FDA review of eteplirsen. Let's first define what accelerated approval means.
When I was young, I remember my parents saying that “ignorance is bliss”. I never liked that expression and it has become more relevant for me when I speak with parents who have children affected by Duchenne muscular dystrophy who are trying to navigate educational or health care systems. Parents are often overwhelmed by the complexity of these programs and their…Continue
Added by Brian Denger on October 4, 2012 at 11:00am — No Comments
The much-anticipated 48-week results on eteplirsen, a drug designed to skip exon 51, were presented today by Sarepta Therapeutics on a webinar and press release. On the webinar, Sarepta’s Chief Executive Chris Garabedian presented data about the dystrophin found in study participants’ muscle biopsies and the…Continue
I ran the 6th Annual Friends and Neighbors in Action 5k in Monroe Township, New Jersey on September 22nd. The race supported Jake and Ben Swomiak, brothers with Duchenne, and Parent Project Muscular Dystrophy. This was my first PPMD race (and I should add that I finished 2ndin my age group; getting old has its benefits)! My professional…Continue
We are so happy to support the work of Dr. Toshifumi Yokota at the University of Alberta, in Canada. Exon skipping looks promising as a viable therapeutic for Duchenne. Dr. Yokota is taking…Continue
Added by Sharon Hesterlee on September 27, 2012 at 2:30pm — No Comments
With Congress decamping from Washington and not expected to return until after the November election, we thought we'd revisit one of the few bright spots of bipartisanship this Congress – the enactment of the latest FDA user fee law (PDUFA), known as the FDA Safety and Innovation Act or FDASIA. (Washington loves a good acronym).
Added by Ryan Fischer on September 26, 2012 at 10:00am — No Comments
Parent Project Muscular Dystrophy’s Women’s project is still in need of moms to participate in the Mothers’ Survey. This survey collects information from mothers of children with Duchenne and Becker, including your needs, your strengths, and your wellbeing. The results from this survey will help us develop new…Continue
Sure, I’ve been there, several times, but it has been a long time, more time than I realized. We actually had no plans to visit, but it seemed worth doing, having just spent the weekend in Dowagiac, Michigan, remembering summers long ago. It was an off-hand idea: my daughters Jenny and Michelle were flying back to New York, from South Bend…Continue
Added by Pat Furlong on September 19, 2012 at 4:00pm — No Comments
Added by Pat Furlong on September 18, 2012 at 11:30am — No Comments
With the 5th annual Coach To Cure MD right around the corner on September 29, 2012, we wanted to tell you why you should make this the year you get in the game and show your support for one of the biggest national awareness campaigns in the Duchenne community. And we wanted you to hear it from families that have participated since the beginning... Here's why the Dumm Family joined the team!
Added by Danielle Garrigan on September 17, 2012 at 2:30pm — No Comments
Fall is the start of a new year for so many of us, in so many different ways. The kids are back at school, the calendar is once again packed, and…Continue
Added by Pat Furlong on September 13, 2012 at 2:00pm — No Comments
Connor Vassigh is 6 ½ years old and his family have been among our most active participants in Coach To Cure MD over the last few years. Connor hosts a “Muscle Party,” inviting friends and family to have fun, party together, and enjoy a Saturday of football. He is a first grader who…Continue
Added by Will Nolan on September 13, 2012 at 1:30pm — No Comments
“Lauren’s Hope” started out as “You Name It,” and began as a fun little business making personalized gifts for women and children. They were approached by Lauren’s Hope for A Cure to…Continue
The end of summer and the arrival of autumn brings up a mix of emotions in both parents and their children. Back to school is exciting, a new start. But it also means the end of summer and the beginning of the flurry of activity – schedules, calendars, multitasking. This month, Colorado…Continue
Added by Pat Furlong on September 13, 2012 at 1:21pm — No Comments
Parents of children with Duchenne and Becker muscular dystrophy often experience a diagnostic odyssey that is too long and too emotionally difficult. Parents who have shared the story of their diagnoses told us:
“My son’s pediatrician blew off my concerns and told me some children just develop slower than others. During the following months I repeatedly…
Added by Holly Peay on September 13, 2012 at 1:12pm — No Comments
Cardiac care and cardiomyopathy remain major areas of interest and concern for all people with Duchenne. While advances in respiratory care have improved respiratory outcomes, dilated cardiomyopathy and heart failure remain the leading cause of death in Duchenne.…Continue
Added by Kathi Kinnett on September 13, 2012 at 12:00pm — No Comments