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All Blog Posts Tagged 'PPMD Blog' (812)

FDA’s Patient Focused Drug Development Initiative – Request for Public Comment

PPMD and other groups submit joint statement making the case for neuromuscular disorders affecting children

As many advocates know from earlier blog postings, PPMD has been deeply and passionately engaged to enhance how the Food and Drug Administration (FDA) goes about its business of reviewing potential therapies and treatments for patients with Duchenne. Our work is focusing on ensuring that the agency moves as expeditiously as possible and…


Added by Ryan Fischer on November 15, 2012 at 11:00am — No Comments

Changing What's Next

Being a runner, I have found, has helped me on the job.  Running races, achieving goals, and managing projects all require a similar ability to stay disciplined and focused. Organizing a successful event and finishing a race both provide a tremendous sense of accomplishment and ego boost. I have also found that running offers a unique problem-solving opportunity for the other “events” I’m running. It is the only time of…


Added by Mary G. Connolly on November 15, 2012 at 11:00am — No Comments

Duchenne Drug Development Round Table

There are a lot of frustrations in the Duchenne community—one of the most common themes I’ve heard is “why isn’t all this research and drug development coordinated better?” The problem, of course, is that there are a lot of companies and funding groups trying to help at once—sometimes it may feel like too many…


Added by Sharon Hesterlee on November 15, 2012 at 10:00am — 5 Comments

Representing Duchenne at the Congressional Rare Disease Caucus Briefing

Today, Parent Advocate and Founder of Two Smiles One Hope Foundation, Alison Willis and I represented the Duchenne community at the Congressional Rare Disease Caucus Briefing in Washington, DC. Alison did a remarkable job speaking on behalf of PPMD regarding the Patient Participation in Medical Product Discussions Provision within the newly passed…


Added by Ryan Fischer on November 14, 2012 at 3:00pm — No Comments

What is Duchenne? (Two Moms, Co-Blogging)

Ivy's son, Rain


What IS Duchenne?

by Pat Furlong


Last year, Ivy and I agreed to write a co-blog. I would introduce Ivy’s or wrap something…


Added by Pat Furlong on November 13, 2012 at 8:30am — 2 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…


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

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…


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

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Wow! What an unbelievable few weeks we have had in this community! Never in the 18…


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…


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…


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…


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).



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…


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…


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…


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…


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…


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

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