All Blog Posts Tagged 'PPMD Blog' (819)

Management of End Stage Heart Failure: Ventricular Assist Devices (VAD)

In light of PPMD’s Cardiac Webinar Series, our Director of Care, Kathi Kinnett answers questions/concerns about Ventricular Assist Devices (VAD).


There has been recent discussion worldwide…


Added by Kathi Kinnett on October 11, 2012 at 2:30pm — No Comments

Better, Faster, Now!

These are the words that best describe PPMD’s research plan.

  1. We aim to put better drugs into development for clinical trials because we can’t afford the failures.
  2. We apply funding strategically to increase the speed of testing new drugs.
  3. We prioritize and fund the testing in Duchenne of drugs that are already approved for other purposes.

Everything we do goes through this filter to make sure that we are achieving one of these…


Added by Sharon Hesterlee on October 11, 2012 at 11:00am — 4 Comments

Positive Phase 1 trial results for SMT C1100 in Duchenne (Summit PLC)

Following closely on the heels of the good news about Sarepta’s exon 51 skipping strategy, PPMD is pleased to report that Summit…


Added by Sharon Hesterlee on October 10, 2012 at 10:30am — 13 Comments

Webinar Q&A: Monitoring Heart Disease in Duchenne and Carrier Moms/Daughters

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.

Topics Included:

  • Screening of patients…

Added by Kathi Kinnett on October 9, 2012 at 3:30pm — No Comments

Understanding Accelerated Approval

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.


Because it…


Added by Ryan Fischer on October 5, 2012 at 2:00pm — 1 Comment

Protecting family caregivers from employment discrimination

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…


Added by Brian Denger on October 4, 2012 at 11:00am — No Comments

Promising News from Sarepta on Exon 51 Skipping Trial

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…


Added by Holly Peay on October 3, 2012 at 4:00pm — 5 Comments

How I became a runner

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…


Added by Mary G. Connolly on October 3, 2012 at 12:00pm — 2 Comments

Parent Project Muscular Dystrophy awards $50,000 to Dr. Toshifumi Yokota for multi-exon skipping project

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…


Added by Sharon Hesterlee on September 27, 2012 at 2:30pm — No Comments

Implementing PDUFA

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

Calling All Mothers - We Need Your Input!

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…


Added by PPMD on September 25, 2012 at 8:50am — 1 Comment

Notre Dame

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…


Added by Pat Furlong on September 19, 2012 at 4:00pm — No Comments

Why we joined the team: The Dumm Family

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

September note: Back to School

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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…


Added by Pat Furlong on September 13, 2012 at 2:00pm — No Comments

My Voice: Connor Vassigh, 6 ½ years old

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…


Added by Will Nolan on September 13, 2012 at 1:30pm — No Comments

Medical Alert Bracelets for Duchenne

For several years now, there has been the question of, “Where is the best place to get medical bracelets” and “what should they say?”  Finally, I think we have an answer.


“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…


Added by Kathi Kinnett on September 13, 2012 at 1:30pm — 11 Comments

Falling into Fall (Two Moms, Co-Blogging)

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…


Added by Pat Furlong on September 13, 2012 at 1:21pm — No Comments

Early diagnosis makes a difference

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

Announcing PPMD's Cardiac Webinar Series

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.…


Added by Kathi Kinnett on September 13, 2012 at 12:00pm — No Comments

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