All Blog Posts Tagged 'PPMD Blog' (819)


Staff
Giving Thanks, Looking Forward

Every Thanksgiving, I find myself with a lot to be thankful for: my family’s love and support, our Duchenne community, and your tireless efforts.



This year is no exception. We’ve accomplished so much together. When I think of all that our community has made possible over the years, all that we’ve done in 2012, and all that we’re doing today, my heart fills with…

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Added by Pat Furlong on November 20, 2012 at 2:00pm — 2 Comments


Staff
Help Protect Federal Spending on Rare Diseases like Duchenne

PPMD Taking Action to Avert Sequestration



Those engaged in federal advocacy probably thought the Prescription Drug User Fee Act and its acronym PDUFA would take the cake…

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Added by Ryan Fischer on November 19, 2012 at 4:57pm — 2 Comments

Congratulations & Thank You

Congratulations to this year’s winner of the American Public Health Association Disability Section Lifetime Achievement Award, Dr. Gloria Krahn, the Director of the Division of Human Development and Disability at the Centers for Disease Control and Prevention’s (CDC’s) National Center on Birth Defects…

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Added by Holly Peay on November 15, 2012 at 12:20pm — No Comments


Staff
November Note: Weathering the Storm

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So many of our friends and family, including the…

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Added by Pat Furlong on November 15, 2012 at 12:00pm — No Comments


Staff
Transforming Duchenne Care Update: Part II - Meeting with the Cystic Fibrosis Foundation

It’s been a little while since our last update, but I wanted to wait until I had definite news. It’s been an exciting month for Transforming Duchenne Care (TDC), and I will do my best to get everyone up to speed. We had two important and informative meetings. Below is an update on our recent meeting with the Cystic Fibrosis Foundation, as well as PPMD’s contribution to Duchenne care.…

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Added by Kathi Kinnett on November 15, 2012 at 12:00pm — No Comments


Staff
My Voice: Michael Counterman, 20 years old

Michael Counterman is a 20-year-old at the University of Massachusetts in Boston. He has worked with PYD, an inclusion-based organization that helped him interact with other kids and raise awareness about Duchenne. Recently recognized for his ongoing contribution to the group, Michael is an inspiration to…

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Added by Will Nolan on November 15, 2012 at 11:30am — No Comments


Staff
Transforming Duchenne Care Update: Part I - Meeting with the MDA

It’s been a little while since our last update, but I wanted to wait until I had definite news. It’s been an exciting month for Transforming Duchenne Care (TDC), and I will do my best to get everyone up to speed. We had two important and informative meetings. Below is an update on our recent meeting with the MDA. Click here to read about our…

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Added by Kathi Kinnett on November 15, 2012 at 11:30am — No Comments


Staff
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…

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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…

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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…

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


Staff
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…

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


Staff
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…

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


Staff
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…

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


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

Background 

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…

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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…

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


Staff
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…

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


Staff
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…

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


Staff
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…

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


Staff
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…

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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…

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

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