All Blog Posts Tagged 'Staff' (198)

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

Living Duchenne: I Love the Internet 

Dear Internet, how I do love thee!…

On January 24, PPMD hosted the third in our series of four cardiac webinars. This webinar was dedicated to the subject of Ventricular assist device (VAD) use in patients with Duchenne. We were pleased to be joined by a panel of four outstanding surgeons who are all experts in the field of cardiothoracic surgery.

The four…

As you know, we head into an important year of Advocacy with the muscular dystrophy community determined to Reauthorize the MD-CARE Act. For the past few months PPMD, FED, and MDA have been working…

On October 23, 2012, PPMD hosted a webinar with Sarepta to discuss of the 48 Week dystrophin and 6-minute walk test (6MWT) data from the Phase IIb extension study in Duchenne as well as next steps for the eteplirsen program. Chris Garabedian, President and CEO of Sarepta…

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…

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…

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…

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.…

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 […

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…

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

Living Duchenne: My…

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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…

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…

The 194^th 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…

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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…

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,…

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…

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…