August 2014 Blog Posts (9)

Let’s improve DuchenneConnect’s informed consent. Give your opinion by September 7th.

You may be thinking, “An informed consent document? No way do I want to read one of those!” But you can demonstrate the power of our community and help us write a more understandable, responsible informed consent for our community’s registry.

As many of you know, DuchenneConnect was…


Added by Holly Peay on August 28, 2014 at 11:25am — No Comments

Run Big Sur or Star Wars to End Duchenne!

Two NEW and exciting races have been added to our Run For Our Sons calendar for 2015....which adventure will you…


Added by Nicole Herring on August 19, 2014 at 1:30pm — No Comments

PPMD Submits Cardiac Recommendations to FDA

As you know, PPMD recently facilitated a multi-stakeholder process involving patients, parents, investigators, industry, and other interested parties which culminated in a draft guidance document “Guidance for Industry:  Duchenne Muscular Dystrophy,…


Added by Pat Furlong on August 18, 2014 at 1:00pm — No Comments

MD-CARE Act: We are not done yet! Tell the Senate to act this September.

Sure, we’re in the lazy days of summer and Congress is on recess, but now is not the time to slow down on the MD-CARE Act. Let’s keep our momentum going. Take a minute to thank the House for passing the bill and tell the Senate why we need them to take up the…


Added by Ryan Fischer on August 13, 2014 at 9:30am — No Comments

The Breath of Life: New 3-Part Pulmonary Webinar Series

The lungs require the strength of many muscles to help a person to breathe and cough. As people with Duchenne grow older, the respiratory muscles, like the skeletal muscles, become weaker. Like the heart, every person with…


Added by Kathi Kinnett on August 12, 2014 at 9:30am — No Comments

Sarepta Update: August 7, 2014

Yesterday Sarepta released its second quarter earnings report and included an update on their Duchenne muscular dystrophy program. We asked Sarepta to provide the community with a written update regarding their exon 51 confirmatory…


Added by PPMD on August 8, 2014 at 9:00am — No Comments

Duchenne in Paradise

Every year I try to escape the real world for a couple of days and retreat with my family to probably my favorite place on Earth – Dowagiac. I’ve reminisced before, but this place is magical to me. It is my childhood, it is summer, it is magic. With so much going on in our community – thankfully! – it has been harder and harder to get away. But I wanted to…


Added by Pat Furlong on August 7, 2014 at 10:30am — 1 Comment

Welcoming Drs. Dongsheng Duan & Jill Rafael-Fortney to PPMD's Scientific Advisory Committee

We are excited to welcome the two newest members of our Scientific Advisory Committee, Dongsheng Duan, PhD and Jill Rafael-Fortney, PhD! Our Scientific Advisory Committee, chaired by PPMD Senior Scientific Advisor Dr. Lee Sweeney, includes leading experts in basic and clinical Duchenne research and Duchenne clinical management. The committee expertly reviews every grant that comes in and offers funding or advice on…


Added by PPMD on August 6, 2014 at 11:30am — 1 Comment

Remembering Scott – In his own words

I had no words for Scott’s death. In fact, he had been actively dying for several years. I had hoped that he might see Rebekah for one last time, though I realized they were both too sick and living in different countries made it impossible. The good news is that many people with Duchenne never find a love.…


Added by Pat Furlong on August 4, 2014 at 10:30am — No Comments

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