January 2015 Blog Posts (15)

Parent Project Muscular Dystrophy Awards UCLA $50,000 Exploratory Grant for Evaluation of Exon Skipping Enhancers in Duchenne

PPMD announced today that they will award Dr. M. Carrie Miceli and her team at UCLA’s David Geffen School of Medicine and College of Letters and Science, a $50,000 exploratory grant to continue their evaluation of exon skipping boosters for the treatment of Duchenne. 


Duchenne muscular dystrophy is the most common fatal genetic disorder diagnosed in…


Added by PPMD on January 29, 2015 at 1:00pm — 2 Comments

Surviving Blastomycosis at the age of 20

Hello everyone; I haven't blogged on here since the new web site as life has not stopped or slowed down for us. I have a son Casey who's birthday is today and he is 21 and has Duchenne MD. He recently was diagnosed with Blastomycosis which is a fungal disease that was growing in his left lung. We came close to losing him as diagnosing Blasto is not an easy task. It tends to present itself as lots of other illnesses and for many by the time it is discovered it is too late. Once again we were… Continue

Added by Renee Rivard-Gould on January 29, 2015 at 11:53am — No Comments

Get Active in Advocacy

2014 was a historic year in our community. 2015 promises to be another critical year in Duchenne and we need you to help keep the momentum going! Just look at the ‘to-do’ list:

  • MD-Care Act amendment implementation begins
  • New Drug Applications submissions expected by several companies
  • The FDA has stated publicly…

Added by Ryan Fischer on January 28, 2015 at 1:30pm — No Comments

FDA Acknowledges the Importance of Benefit-Risk Preferences: Why this Matters to Duchenne

This started as a blog about the second publication on PPMD’s benefit/risk study, which was published in late December in a journal called The Patient (and more on that later). But something remarkable happened this week that helps frame…


Added by Holly Peay on January 26, 2015 at 10:23am — No Comments

Important Vaccinations Recommendations

Vaccinations are very, very important to protect us against infection and disease – but they are also very, very confusing. 

Some vaccines are “live,” some are “attenuated (killed),” some protect against viral infections, some protect against bacterial infections, some are…


Added by Kathi Kinnett on January 23, 2015 at 12:00pm — No Comments

BMS & The Voice of the Patient

For the last two years, global biopharmaceutical company Bristol-Myers Squibb (BMS) has organized a day when all of their drug development project teams from across the world would have the opportunity to take some time to listen to patients and their caregivers. Wednesday was that…


Added by Pat Furlong on January 22, 2015 at 10:30am — 1 Comment

Student needs help with inclusion survey

Dear Friends,

high school student (Caleb SIzemore) is asking for help with a project about inclusion. 

Caleb is doing a senior thesis on avenues to increase social support for boys with Duchenne, based on his experience living with Duchenne and on research. He needs more responses to finalize the project. 

To participate…


Added by Ryan Fischer on January 20, 2015 at 6:38pm — 1 Comment

No Choice...Part One

This is my first post to my new blog. I have another blog named “dadsfavoriteword” that has given me the chance to share my life’s experiences as a dad, husband and friend using one word that seems to exemplify what I am going through at any one time. This blog however, will certainly be more raw and emotional than I am used to writing. This blog concerns a teenage boy who is not my biological son, but a son that I chose to have 11 years ago. His name is Charlie.

Although a lot of…


Added by Kurt Main on January 16, 2015 at 12:44pm — 2 Comments

Meet our 2015 PPMD Adult Advisory Council!

“We were the generation that was hoped for… And now we’re here.” L. Vance Taylor

The PPMD Adult Advisory Council (PAAC) serves to ensure that the voices of adults living with Duchenne and Becker muscular dystrophy are reflected in…


Added by Annie Kennedy on January 15, 2015 at 1:00pm — No Comments

Where the Long, Winding Path has Led

I sit here in my home office on a snowy January day in Maryland, and I think about where I am at now in life and how I got here. As a naïve, newly minted Ph.D. in 1980, it wasn’t inevitable, or even predictable, that I would ultimately join all of you in the fight to end Duchenne. But, I’m really glad that I’m here and I’ll tell you a bit about the path along…


Added by John D. Porter, PhD on January 12, 2015 at 10:00am — 1 Comment

Sarepta Update: 168 Week Data

This morning Sarepta released the 168 week data on eteplirsen.  

For weeks there has been speculation, guesses, and hope about what we might learn.  We have been following the stories on social media, grateful for every photo or video. We are thrilled that the open label eteplirsen studies (confirmatory study and non ambulatory study) are actively…


Added by Pat Furlong on January 12, 2015 at 9:30am — 3 Comments

PPMD Welcomes New CEO, John Porter, PhD

We're excited to announce that John Porter, PhD, former program director of the National Institutes of Health (NIH)/National Institute of Neurological Disorders and Stroke (NINDS) will take on the role of Chief Executive Officer for Parent Project Muscular Dystrophy.

As program director at the NIH/NINDS, Dr. Porter was…


Added by PPMD on January 12, 2015 at 9:00am — No Comments

Update on the 2015 Flu

As you may have noticed, despite receiving the 2014-2015 influenza (flu) vaccine, many people have gotten the flu.  It turns out that the vaccine contained inactivated (dead) virus that protected against 3 strains of influenza:


  • Influenza B/Massachusetts (2012-like antigen)
  • Influenza A/California (H1N1 – like…

Added by Kathi Kinnett on January 9, 2015 at 10:28am — 6 Comments

Without Endings There Are No Beginnings…

It’s New Year’s Day 2015 and I think Janus, the two-headed Roman God who looks forward and backwards at the same time, is a fitting representation of my life right now. I need to tell you all that I have had to make a difficult decision this year—at the end of this month I will be winding up my time at PPMD while simultaneously starting mid-month with the Myotonic Dystrophy…


Added by Sharon Hesterlee on January 5, 2015 at 1:30pm — 1 Comment

Eplerenone FAQs - Early Treatment of Cardiomyopathy in Duchenne

Dr. Subha Raman confers with colleagues in a laboratory at The Ohio State University Wexner Medical Center. Raman published a study that shows a combination of well-established heart medications can dramatically slow the rate of heart damage in patients with Duchenne muscular dystrophy. …


Added by PPMD on January 1, 2015 at 12:30pm — No Comments

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