April 2013 Blog Posts (15)

He Knows.....How I told my son that Duchenne is a fatal condition

Just your not so average Thursday afternoon for a dude with Duchenne and his momma. A conversation that needed to happen, but this momma had been struggling to find the right time to have it.  Dreading revealing a secret that I have been keeping from Jake, but also hiding it was just as dreadful. Sometimes the Universe does not give you a choice and presents you with a golden ticket in the form of an opportune moment. Do you take it? Do you crumple it up and toss it into oblivion you like…


Added by Danielle Bath on April 30, 2013 at 6:00pm — 6 Comments

DuchenneConnect Presents 2012 Data & 2013 Update

Parent Project Muscular Dystrophy and DuchenneConnect are happy to present the 2012 DuchenneConnect Year-End Report! Read about the registry’s accomplishments in 2012 and goals for 2013. We had a busy and exciting year as we…


Added by Ann Martin on April 30, 2013 at 10:55am — No Comments

Introducing Duchenne Central!

I have met some amazingly innovative people in my travels. Many who are dedicated to improving the lives of those living with rare diseases. Wendy White is one of those people. Wendy is the founder of Siren Interactive.  Siren is a digital relationship marketing agency focused exclusively on addressing the…


Added by Pat Furlong on April 29, 2013 at 10:00am — No Comments

Report from MDA's Scientific Conference 2013

PPMD President Pat Furlong and Vice President of Research Sharon Hesterlee are attending MDA’s Scientific Conference this week in Washington, D.C. Most of what was presented are results that were previously announced. However, there were some interesting presentations, as well as, results worth repeating, that we wanted to share. Sharon and Pat will continue to blog from the conference:


From Pat Furlong

Interview with Thomas Voit

While the content…


Added by PPMD on April 24, 2013 at 10:00am — 6 Comments

Continued Access to Drisapersen for Patients Completing the US Ph IIb 48-week study

Below is the latest update PPMD has received from Dr. Rohit Batta, Global Medical Affairs and Patient Relations Leader in GSK's Rare Disease Unit :

The purpose of this communication is provide an update to you, as you have requested, on GSK’s plans for providing the option of continued access to drisapersen for boys completing the US Ph IIb 48 week study (DMD114876)…


Added by PPMD on April 24, 2013 at 9:30am — No Comments

Putting Patients First

PPMD is proud to share an advance copy of Putting Patients First, a white paper outlining…


Added by Pat Furlong on April 22, 2013 at 12:00pm — No Comments

Duchenne: An Intergenerational Issue

Guest post by Gail D. McVicker, Grandmother of Jake (10) and Ryan (6) - Pennsylvania

Duchenne: An Intergenerational Issue

Five years ago, I did not know the world of Duchenne muscular dystrophy existed. Before my young grandsons were officially diagnosed in 2008, my daughter told me that some genetic…


Added by PPMD on April 17, 2013 at 2:49pm — 2 Comments

My Voice: Matt Scheivert, 17 years old

As you know, each month we feature someone in the community with Duchenne, that has done something really special to raise awareness and help us in our fight. This month, we are featuring 17-year-old Matt Scheivert, who doesn’t have Duchenne, but has already decided that he wants to continue to work in Duchenne research and advocacy. He plans to attend…


Added by Will Nolan on April 17, 2013 at 2:22pm — No Comments

Pipeline Update: Drisapersen, GSK Report from Cold Spring Harbor Conference

PPMD received the email update below from Hans Schikan at Prosensa:

Dear all,

Today, at the Cold Spring Harbor conference in the US, Dr John Kraus of GSK gave an excellent presentation on the outcome of the 117 (DEMAND II) clinical trial. The primary endpoint in this exploratory study was met and a statistically and clinically meaningful difference of 35 meters in the six-minute walk test between the continuous dosing regimen of 6mg/kg per week and placebo was…


Added by PPMD on April 12, 2013 at 11:39am — No Comments

Drug Development Prospects for Duchenne: The Pipeline is rich and deep, with lots of forward momentum

This spring brings news of progress to the Duchenne community as Sarepta engages in talks with the Food and Drug Association (FDA) to determine if they agency will review an application for accelerated approval for the exon 51 skipping drug eteplirsen and releases its 74 month data…


Added by Sharon Hesterlee on April 9, 2013 at 9:00pm — 5 Comments

Pipeline Update: Drisapersen, GSK

Below is the latest update PPMD has received from Dr. Rohit Batta, Global Medical Affairs and Patient Relations Leader in GSK's Rare Disease Unit :

To keep you updated on the progress of the drisapersen (previously GSK2402968) clinical development program, as you have requested, I am writing to inform you that the results of the GSK Phase IIb study investigating drisapersen as…


Added by PPMD on April 5, 2013 at 10:51am — No Comments

Living Duchenne: Silence and Noise

Guest post by Ivy Scherbarth. Ivy is a Colorado/Wyoming FACES Coordinator for PPMD. Follow Ivy at her blog, Living Duchenne.

Living Duchenne: Silence


One interesting thing about silence is listening to…


Added by PPMD on April 4, 2013 at 11:30pm — No Comments

Ryan Kaplan – in memory

Funeral dirge. Hearts break so many times, it is impossible to keep track.  A tidal wave of memories, nearly too much to bear. Minds racing, reviewing the journey that was Duchenne and all of the ‘what ifs’ and ‘if only’ that remain. Ryan’s father, mother, sisters – raw and alone, returning to the home once overflowing with Ryan and now empty.

I met Howard Kaplan many years ago in Chicago. He is a tall, broad shouldered man and in the business of protection.  His company,…


Added by Pat Furlong on April 4, 2013 at 11:24pm — No Comments

NH Governor Maggie Hassan to Declare Friday, April 5th- Duchenne Muscular Dystrophy Awareness Day

A Call to Action for all NH Constituents!

Seacoast, New Hampshire- 8th grader Rebecca Gowing wanted to make a difference in her community. After learning that her sister’s classmate and friend, Lucas Currier, had Duchenne Muscular Dystrophy she took action to help raise funds and spread awareness, creating Art for A Cure…


Added by Ryan Fischer on April 2, 2013 at 9:00pm — No Comments

PPMD's 2013 Annual Connect Conference - Duchenne: Better, Faster, Now!

Hello Duchenne community!

Summer is just around the corner and with that comes sunshine, fireflies and PPMD’s Annual Connect Conference.

We are excited to be back in Baltimore, MD at the beautiful Marriott Waterfront this year and really excited about this year’s program.

In 2013…


Added by Kimberly Galberaith on April 2, 2013 at 11:30am — No Comments

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