All Blog Posts Tagged 'Staff' (198)

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

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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…

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)…

PPMD is proud to share an advance copy of Putting…

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…

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…

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…

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…

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…

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…

On March 26, PPMD President Pat Furlong, along with other members of the Duchenne community (internationally, including those like Pat that traveled from the U.S.) met with the UK's Shadow Minister of Health, Andrew Burnham in a meeting at Parliament.

The Video

Setting the Stage

The meeting opened with a video, that of a 4-year-old boy, Jack, diagnosed in…

Parent Project Muscular Dystrophy (PPMD) has awarded University of Minnesota researcher Dr. Rita Perlingeiro a grant for $220,000 to develop a therapeutic approach for Duchenne using a new type of "adult-derived" stem cell called "induced pluripotent stem cells" or "iPSCs." iPSCs can be created from non-controversial sources like skin, have the ability to multiply indefinitely, and can be coaxed to become many different kinds of tissue types.



Previously, Dr.…

• First patient clinical trials of SMT C1100 expected to start H2 2013
• Summit to showcase program at international scientific conference

Summit's Press Release:

Summit today outlines its future plans for the continued development of utrophin modulators for the treatment of DMD. Utrophin modulation is a disease modifying approach that has the potential to treat all genetic forms of DMD. Summit’s…

As we remind you to register for DuchenneConnect or…

Help Bring Run for Our Sons to your own backyard.

I hear it all the time, “I want to do a fundraiser but not sure what to do or where to start” – I ask “have you thought of doing a 5K or fun run in your community?” – The rest is Run for Our Sons history!

5Ks and fun runs are fast becoming one of PPMD’s…

We were thrilled that 119 parents/guardians were willing and able to complete the entire treatment preferences survey. We are analyzing the data. We are doing the first part of the data analysis in-house, and the second part with our health economist collaborator. Our plan is to present this information to the FDA, to industry and clinical…

When Chris and Patrick died, there was no Internet, no way to connect. The days just after they died, life was busy but at the same time heartbreaking – there was so little time to think about anything. After all was over, silence settled in. We were left with the burden of living without them, of going on and trying to make some sense of it.

No one called, not…

Parent Project Muscular Dystrophy, The Muscular Dystrophy…

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

PTC Therapeutics, Inc. (PTC) today announced the successful completion of a $60 million financing led by Brookside Capital Partners Fund, L.P.

"This substantial financing enables PTC to continue to pursue the regulatory approval and delivery of ataluren to patients," stated Michael Schmertzler, Chairman of the Board. "PTC's Board of Directors welcomes the breadth of support and expertise of our new investors."…