PPMD Community

November 2012 Blog Posts (16)


Staff
Recent Petition to the FDA

Duchenne is getting a great deal of attention these days. With the excitement around the Sarepta trial and more trials coming online, more attention to Duchenne is a great thing as we continue to raise awareness. Personal stories about families only help to educate the public on Duchenne. It allows them to relate and perhaps to think, If this was my son, what would I do??

 

A recent…

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Added by Ryan Fischer on November 27, 2012 at 4:00pm — No Comments

Developing a Risk Tolerance Study for FDA

We, the Duchenne community, have entered into a new era where more trials are coming online and the possibility of treatments becoming a reality. PPMD remains focused on working with the FDA to inform them about Duchenne so they can better understand the complexities of the disease and its patient population.



We have been working hard to create a survey about perceptions of benefits and risk tolerance of parents/guardians of individuals with Duchenne,…

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Added by Holly Peay on November 21, 2012 at 10:37am — No Comments


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
Summit: Repeat dosing of SMT C1100 for treatment of Duchenne meets endpoints in Phase 1 trial

News Release:

Summit Corporation plc 

(‘Summit’ or ‘the Company’)



REPEAT DOSING OF SMT C1100 FOR TREATMENT OF DUCHENNE MUSCULAR DYSTROPHY MEETS ENDPOINTS IN PHASE 1 CLINICAL TRIAL 

 

New formulation delivers drug levels that are predicted to significantly increase utrophin…

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Added by PPMD on November 7, 2012 at 10:00am — No Comments


Staff
Duchenne and the Election

Tomorrow, we will head to the polls to choose who will lead our nation for the next four years.



While many of us – particularly those of you who live in key "swing" states that will likely determine the outcome of the election – may be tired of the unceasing television commercials, robocalls and mailers, it's important to recognize that regardless of who is elected, creating…

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Added by Ryan Fischer on November 5, 2012 at 2:30pm — No Comments

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