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All Blog Posts Tagged 'PPMD Blog' (813)

American Taxpayer Relief Act of 2012

Although it’s a New Year, there is still an opportunity to make a charitable donation for the 2012 tax year. The American Taxpayer Relief Act of 2012 that averted the fiscal cliff and maintains incentives for charitable giving for most Americans affects charitable giving in several ways; some still to be determined and we will keep you apprised of any developments for 2013 giving.

One important piece we need to make you aware of now is the return of the IRA charitable…


Added by Mary G. Connolly on January 16, 2013 at 1:50pm — No Comments

Ventricular Assist Device (VAD) use in patients with Duchenne

End stage dilated cardiomyopathy (DCM) is a known complication of Duchenne muscular dystrophy. DCM leads to end stage heart failure and the premature death of patients with Duchenne. Given recent advances in respiratory care, heart failure is the cause of death in more than 40% of patients with Duchenne.


A “ventricular assist device (VAD)” is a mechanical pump that is connected to the heart in order to support its function. The type of device that would be considered in…


Added by Kathi Kinnett on January 16, 2013 at 1:30pm — No Comments

My Voice: Anthony DeVergillo, 19 years old

When you meet Anthony DeVergillo in person, you can’t help but leave the conversation with a smile on your face. This almost 20-year-old is the definition of optimism. In fact, he has made spreading optimism his life’s work. From an early age, Anthony realized he had two choices with his Duchenne diagnosis…


Added by Will Nolan on January 16, 2013 at 1:00pm — No Comments

Your Go-to Duchenne News Resource

The Duchenne community is bigger than ever and Duchenne news is coming faster and more frequently than we could ever have hoped for. 2013 is already promising to be a big year in research, advocacy, care, and raising awareness. But…this can be overwhelming. With so much information coming your way, with a barrage of messages and posts through social media in your face, it’s hard to know what’s legit, what’s truly promising, and unfortunately, what just isn’t true.…


Added by Will Nolan on January 16, 2013 at 1:00pm — No Comments

GSK Update

Update from GSK on the progress of their drisapersen (previously GSK2402968) clinical development program:

To keep you current on the progress of the drisapersen (previously GSK2402968) clinical development program, as you have requested, I am writing to inform you that the last patient has been recruited within the drisapersen US Phase II clinical study […


Added by PPMD on January 10, 2013 at 9:00pm — No Comments

Educating healthcare providers about the need for early diagnosis

Families of children with muscular dystrophy and other muscle disorders often experience a diagnostic odyssey that is too long and too emotionally difficult. PPMD, with support from The Centers for Disease Control and Prevention (CDC), created the National Task Force for Early Identification of Childhood Neuromuscular Disorders to address the continued gap between the time when the first symptoms of muscle weakness…


Added by Holly Peay on January 9, 2013 at 2:00pm — 1 Comment

Living Duchenne: My Year in Review


Guest post by Ivy Scherbarth. Ivy is a Colorado/Wyoming FACES Coordinator for PPMD. Follow Ivy at her blog, Living Duchenne.

Living Duchenne: My…


Added by PPMD on January 7, 2013 at 10:30am — No Comments

My Voice: Mohammed Haider, 23 years old

You recognize the face and the name from this year’s holiday campaign – college graduate, working on his Master’s degree, funny. But who is Mohammed “Mo” Haider? What’s his story? This month…


Added by Will Nolan on December 17, 2012 at 1:55pm — No Comments

End Duchenne Gala: 12.6.12

On December 6, 2012, PPMD held its inaugural End Duchenne Gala in New York City. It was a truly remarkable evening thanks in large part to the organizing committee (Catherine Collins, Anessa Fehsenfeld, Janelle Hester, Vicki Singh, and Paula Zenobio) and a room full of compassionate, generous…


Added by PPMD on December 13, 2012 at 4:30pm — No Comments

Exon Skipping & Improving Industry Communications

The 194th workshop of the European Neuro Muscular Centre (ENMC) met in Naarden, The Netherlands, December 7-9, 2012. The title of the workshop was, “Towards Clinical Application of Antisense-mediated Exon Skipping for Duchenne Muscular Dystrophy – Opportunities and Challenges.”  25 representatives of…


Added by Kathi Kinnett on December 13, 2012 at 12:30pm — No Comments

The Greatest of These

Photo Credit



Added by Pat Furlong on December 13, 2012 at 10:30am — No Comments

Webinar Q&A: Cardiac Interventions

Parent Project Muscular Dystrophy recently hosted the second part of our Cardiac Webinar Seriers with Dr. Larry Markham (Children’s Hospital Vanderbilt, Vanderbilt, TN) and Dr. J. Lynn Jefferies (Cincinnati Children's Hosptial, Cincinnati, OH) to discuss cardiac…


Added by Kathi Kinnett on December 5, 2012 at 4:30pm — 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,…


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

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…


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

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…


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…


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

November Note: Weathering the Storm

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So many of our friends and family, including the…


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

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.…


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

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…


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

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