October 2012 Blog Posts (22)

Today's (October 31st) MD STARnet Webinar Postponed due to Hurricane

Dear Friends,

Due to the recent hurricane we are going to postpone tomorrow's MD STARnet webinar.  

We will be in touch regarding the new date. Sorry for the inconvenience. 


Ryan Fischer



Webinar: MD STARnet

Added by PPMD on October 30, 2012 at 1:00pm — No Comments

3 Easy Ways to Leverage Employee Giving & Make the Most Out of Your Donation

Workplace giving campaigns are convenient and effective ways to raise funds in support of ending Duchenne. And they are underway now!

Let your family, friends, coworkers, and everyone on your email list know that the annual United Way and CFC efforts are underway (look for the 'share' button at…


Added by Will Nolan on October 25, 2012 at 9:00am — No Comments

Partnering with the FDA to accelerate approvals- The question of risk tolerance in the Duchenne community


Friends, we have been actively pursuing a regulatory strategy and have had two meetings with the neurology division at the FDA. We are currently working on the 'to do' list for the community and are trying to understand barriers/obstacles to approval from the FDA side, as well as, from the sponsor side of the issue. We are working a smart, targeted call to action. We have learned through years…


Added by Pat Furlong on October 23, 2012 at 11:30pm — 6 Comments


What kind of fundraisers can we do to raise money for dmd???  Any help would be great...I would love to be involved and help find a cure!!!

Added by Laurie on October 23, 2012 at 3:18pm — No Comments

Prosensa Announces Progress on Exon Skipping Compounds for the Treatment of Duchenne Muscular Dystrophy

New release from Prosensa:

£10m in milestone payments received from GlaxoSmithKline


Leiden, The Netherlands, 23 October 2012 – Prosensa, the Dutch biopharmaceutical company focusing on RNA-modulating therapeutics for rare diseases with high unmet need, has selected clinical candidates for two more compounds for the treatment of Duchenne muscular dystrophy (DMD) and has been granted…


Added by PPMD on October 23, 2012 at 12:35pm — 2 Comments


I am very hopeful for better understanding, astrologically, another heavenly sign, far more important than Mars to study the muscles.

I thank the members of this community, having left Shashi…


Added by Antônio Carlos Costa Lima Vieira on October 22, 2012 at 10:47am — No Comments

Facing reality

1997 may not seem so long ago, but at times it feels like half a lifetime to me. That was the year my family learned Matthew’s motor difficulties were something physical therapy could not correct and the first time we heard the word Duchenne. Everyone’s world changes after diagnosis of a devastating chronic illness and over the years my family and I experienced many…


Added by Brian Denger on October 22, 2012 at 8:00am — 3 Comments

Halos for Muscular Dystrophy~The heart beyond the "facts and definitions"

Hi everyone!

So sorry I haven't shared anything for some time. I have been very busy working on a new project that will promote called Halos for Muscular Dystrophy. I would like to welcome you each to join our online Facebook group: http://www.facebook.com/groups/HalosforMD/




Added by dreama whitlock on October 19, 2012 at 10:19am — No Comments

Full 48-week Phase IIb data from eteplirsen study in Duchenne presented at World Muscle Society

Dr. Jerry Mendell, Director of the Centers for Gene Therapy and Muscular Dystrophy at Nationwide Children's Hospital and principal investigator of the Phase IIb eteplirsen study, presented the full set of 48-week data from…


Added by PPMD on October 16, 2012 at 9:00am — No Comments

October Note: Better, Faster, NOW

Note: Sign up to receive updates like this in your inbox every month.


Wow! What an unbelievable few weeks we have had in this community! Never in the 18 year history of PPMD has the Duchenne community been so ripe with…


Added by Pat Furlong on October 15, 2012 at 3:00pm — No Comments

My Voice: Ryan “Wheelz” Schmidt, 20 years old

Wheelz (a/k/a Ryan Schmidt) is a 20-year-old musician from Austin, Texas. He is an accomplished rapper, and with his family, hosts Ryan’s Day – one of the largest annual fundraising and awareness events for Duchenne in Austin, just held last week. The combination of his inspirational raps and Ryan’s Day remind people outside the community that we are in a fight to end…


Added by Will Nolan on October 15, 2012 at 11:00am — No Comments

Virtual Moment of Silence (Two Moms, Co-Blogging)

This month, instead of a pair of essays, Ivy Scherbarth and I are calling for a Virtual Moment of Silence. We invite you, our Duchenne/Becker Muscular Dystrophy community, to use the comments area here to list the names of the loved ones you remember and miss. We honor all…


Added by Pat Furlong on October 15, 2012 at 11:00am — 5 Comments

ImagingDMD: Making muscle biopsies a thing of the past

In the clinical trial world, there are some things that divide us these days- most specifically, what mutation type your son has. However, we are united in wanting better ways to look for the benefits of potential drugs; who isn’t hoping for a day when the muscle biopsy is a relic from the past? 

There are several dedicated research teams doing exciting work…


Added by Holly Peay on October 13, 2012 at 10:00am — No Comments

Management of End Stage Heart Failure: Ventricular Assist Devices (VAD)

In light of PPMD’s Cardiac Webinar Series, our Director of Care, Kathi Kinnett answers questions/concerns about Ventricular Assist Devices (VAD).


There has been recent discussion worldwide…


Added by Kathi Kinnett on October 11, 2012 at 2:30pm — No Comments

Better, Faster, Now!

These are the words that best describe PPMD’s research plan.

  1. We aim to put better drugs into development for clinical trials because we can’t afford the failures.
  2. We apply funding strategically to increase the speed of testing new drugs.
  3. We prioritize and fund the testing in Duchenne of drugs that are already approved for other purposes.

Everything we do goes through this filter to make sure that we are achieving one of these…


Added by Sharon Hesterlee on October 11, 2012 at 11:00am — 4 Comments

Positive Phase 1 trial results for SMT C1100 in Duchenne (Summit PLC)

Following closely on the heels of the good news about Sarepta’s exon 51 skipping strategy, PPMD is pleased to report that Summit…


Added by Sharon Hesterlee on October 10, 2012 at 10:30am — 13 Comments

Webinar Q&A: Monitoring Heart Disease in Duchenne and Carrier Moms/Daughters

Parent Project Muscular Dystrophy recently hosted the first part of our Cardiac Webinar Seriers with Dr. Linda Cripe (Nationwide Children’s Hospital, Columbus, OH) and Dr. Kan Hor (Cincinnati Children's Hosptial, Cincinnati, OH) to discuss monitoring heart disease in Duchenne and carrier moms/daughters.

Topics Included:

  • Screening of patients…

Added by Kathi Kinnett on October 9, 2012 at 3:30pm — No Comments

Understanding Accelerated Approval

With the recent news of Sarepta's promising 48-week data, many have asked how accelerated approval may play into the FDA review of eteplirsen. Let's first define what accelerated approval means.


Because it…


Added by Ryan Fischer on October 5, 2012 at 2:00pm — 1 Comment

Protecting family caregivers from employment discrimination

When I was young, I remember my parents saying that “ignorance is bliss”. I never liked that expression and it has become more relevant for me when I speak with parents who have children affected by Duchenne muscular dystrophy who are trying to navigate educational or health care systems. Parents are often overwhelmed by the complexity of these programs and their…


Added by Brian Denger on October 4, 2012 at 11:00am — No Comments

Promising News from Sarepta on Exon 51 Skipping Trial

The much-anticipated 48-week results on eteplirsen, a drug designed to skip exon 51, were presented today by Sarepta Therapeutics on a webinar and press release. On the webinar, Sarepta’s Chief Executive Chris Garabedian presented data about the dystrophin found in study participants’ muscle biopsies and the…


Added by Holly Peay on October 3, 2012 at 4:00pm — 5 Comments

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