May 2016 Blog Posts (10)

PPMD’s #EverySingleOne Tour: Salt Lake City

With a successful ESO tour stop in Baltimore under our belts, PPMD visited Salt Lake City this past weekend! There’s a special sense of camaraderie that exists when people gather in a single space with a single…


Added by Kathi Kinnett on May 26, 2016 at 3:00pm — No Comments

Buddy's Elegy for Ben Cumbo

Buddy’s Elegy for Ben


Every day of my life, I think of you,

And when I blow my horn, I think of you.

If I could say how much you meant to me,

I have no words to express my love for you.

Your music plays inside my head,

In every song I ever wrote,

This is part of me, I send to you,

It’s coming from my heart,

And every day I think of you.

Right from the sky, I feel your…


Added by Buddy Cassidy on May 25, 2016 at 1:55pm — No Comments

Can I get a taxi? Can I get a witness?

Can I get a taxi? Can I get a witness?


When you’re disabled, your transportation options aren’t great. In this piece, I don’t have any real solutions to the problems I bring up, but I’d like to at least start a conversation. And who knows, maybe someone out there might have some advice for me. Okay, so I’ll go through the various scenarios and the problems that each presents.


I drive

I use the strikethrough to indicate this…


Added by Buddy Cassidy on May 25, 2016 at 1:21pm — No Comments

Sarepta Announces FDA Will Not Complete the Review of the Eteplirsen NDA By The PDUFA Date

The FDA has notified Sarepta that they are continuing their review of eteplirsen and will not be able to complete their work by the Prescription Drug User Fee Act (PDUFA) goal date of May 26, 2016.…


Added by PPMD on May 25, 2016 at 12:00pm — No Comments

PPMD Names Children’s Mercy Hospital in Kansas City, MO, Certified Duchenne Care Center

PPMD Awards Eleventh Clinic Certification as Part of Effort to Ensure People with Duchenne Receive Optimal Care


Children’s Mercy Hospital (Children’s Mercy) in Kansas City, Missouri, became the 11th clinic named…


Added by PPMD on May 16, 2016 at 2:08pm — No Comments

DRSC Definitions

A couple of months ago we posted a write up about some of the efforts PPMD is undertaking to help improve clinical trial design.…


Added by Abby Bronson on May 16, 2016 at 8:40am — 2 Comments

Next Steps with Newborn Screening

For more than two years, PPMD has been leading a national effort to build a newborn screening infrastructure for Duchenne in the U.S.

Led by PPMD's Annie Kennedy, Michele Lloyd–Puryear, M.D., Ph.D., Newborn Screening Consultant for PPMD, and Jerry Mendell, MD of Nationwide Children's, the effort includes more than 50 of the worlds top newborn screening…


Added by PPMD on May 16, 2016 at 8:30am — No Comments

PPMD Brings Together Experts to Assess Steroid Effects on Bone Health in Duchenne

Bone health has long been a confusing and often neglected aspect of Duchenne care. There is very little knowledge of underlying bone health in Duchenne, which is…


Added by Kathi Kinnett on May 12, 2016 at 9:00am — No Comments

Every single carrier.

I am a genetic carrier of Duchenne. My mother was not. This means I was one of the 1:4600 females born with this genetic mutation. As a child, I had muscle cramps, ‘growing pains’ my parents would say. And from those ‘growing pains,’ 20 years later, my sons were diagnosed and I first learned that I was a carrier.


All mothers in our community…


Added by Pat Furlong on May 9, 2016 at 10:07am — 4 Comments

Update from April 2016 Muscular Dystrophy Coordinating Committee (MDCC) Meeting

It's been a rough few weeks for this community, but our efforts to shape and reshape access barrier issues for our community continue -- and last Wednesday we took a giant leap forward. It was a pivotal day that I was proud to be a part of!

The federal intra-agency Muscular Dystrophy Coordinating Committee (MDCC) held its…


Added by Annie Kennedy on May 3, 2016 at 10:28am — 1 Comment

Monthly Archives















Need help using this community site? Visit Ning's Help Page.



© 2021   Created by PPMD.   Powered by

Badges  |  Report an Issue  |  Privacy Policy  |  Terms of Service