PPMD Community

July 2013 Blog Posts (12)


Staff
MD-CARE Act: Is your member in a target state or district?

UPDATED 2/11/14

As we move into the start of 2014 and the February Advocacy Conference we are hopeful to gain momentum to move MD-CARE Act to committee action this year. With 78 cosponsors in the House and 17 in the Senate, we need your help to up our…

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Added by Ryan Fischer on July 31, 2013 at 9:30am — No Comments

Moving back to the USA,

My husband is getting out of the military, therefore we will be relocating to the states. We have 2 children with DMD and are looking for the best place to settle-doctors, schools, community, etc. Please help!

Added by Callaway on July 27, 2013 at 9:33pm — 1 Comment


Staff
Sarepta Therapeutics announces plans to submit New Drug Application to FDA for Eteplirsen – A significant step for the Duchenne community

Today is a landmark day for the entire Duchenne community. Not too long ago, the Duchenne therapy landscape was a barren field with little to no life. Thanks to a commitment to our patients and the field by the U.S. government, international governments, the venture community and industry, this landscape has changed markedly to the point where today we have the announcement for a…

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Added by Pat Furlong on July 24, 2013 at 11:00am — 1 Comment

Steroid use

anyone have any experiences using steroids with their son???  My son is 5 and they r thinking it might b time...not sure how I feel??  help!!

Added by Laurie on July 22, 2013 at 1:05pm — 4 Comments


Staff
Advocating for Open Access

In 2008, PPMD joined the Genetic Alliance and others in the rare disease community, in support of “open access” – essentially ensuring that all NIH funded researchers make available to the public published peer-reviewed manuscripts and articles on a searchable website like PubMed Central or the National Library of Medicines. Making them freely accessible to the public within 12 months of publication.

 …

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Added by Ryan Fischer on July 18, 2013 at 4:30pm — No Comments


Staff
My Voice: John Owen Dumm, 9 years old

For six years now, the Dumm family in North Royalton, Ohio has been hosting JOA Picnic in the Park to raise money and awareness for Duchenne research. This is just one of the many things John Owen's Adventure, Inc. (JOA) does for our community. At the heart of JOA is 9-year-old John Owen Dumm who is a rising fourth grader with Duchenne and an active participant in his namesake event. In fact, John Owen and JOA have…

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Added by Will Nolan on July 16, 2013 at 3:03pm — No Comments


Staff
7.9.13: Update from Congressional Briefing - TAKE ACTION

A big day for Duchenne – Take action to help make it even more impactful!

 

I had the pleasure to join other Duchenne advocates in Washington this week in an effort to bring attention to the need to accelerate treatments for Duchenne, highlighting eteplirsen as a prime example of a promising potential treatment for a subset of the Duchenne population.

A briefing was held at the Rayburn House Office Building, sponsored by Congressman Keating and…

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Added by Ryan Fischer on July 12, 2013 at 10:57am — No Comments


Staff
7.9.13: Update from PPMD/FDA Meeting

July 9, 2013.  

PPMD met with Dr. Janet Woodcock and leadership from the Office of New Drugs, Medical Policy and the Division of Neurology Products, as well as, staff from the Office of Strategic Programs, Office of Health and Constituent Affairs.   The purpose of the meeting was to continue our efforts to provide the agency relevant information and data about Duchenne that will encourage/enable them to be flexible in their review process.  

We…

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Added by Pat Furlong on July 12, 2013 at 10:39am — No Comments

A day to celebrate God's gift of having a son with DMD

What a great day to celebrate life and enjoy all the wonderful things life has to offer. I can sit here and cry and feel sorry for what the future holds for my son who has Duchenne's MD, and for all the family, but I realized that I need to wipe my tears and continue to fight for my son and give him all the love in the world. Who knows what the future holds? I do know that this is such a beautiful gift that God has given me and I will love him more every day.

Added by sima perez on July 8, 2013 at 4:02pm — 3 Comments


Staff
July 9, 2013: One Voice from Capitol Hill to the FDA

Tomorrow, Tuesday July 9th, is a big day for Duchenne advocacy. Advocates will be speaking about the high unmet medical need and urgency for treatments from the halls of Congress to the offices of the FDA.



PPMD will be meeting with individuals from FDA to include CDER, Division of Neurology, Office of Strategic Programs and Staff from the Office of…

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Added by Ryan Fischer on July 8, 2013 at 10:54am — 3 Comments


Staff
Talking About Duchenne

Guest post by Ivy Scherbarth. Ivy is a Colorado/Wyoming FACES Coordinator for PPMD and mom to Hazel, age 8, and Rain, age 6. Rain has Duchenne muscular dystrophy. Follow Ivy at her blog,…

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Added by PPMD on July 5, 2013 at 7:00am — 3 Comments


Staff
Meeting with the FDA: Our topics for discussion

On July 9, PPMD will be meeting with individuals from FDA to include CDER, Division of Neurology, Office of Strategic Programs and Staff from the Office of health and Constituent Affairs.



The topics for discussion will include a review of PPMD’s Board Policy, developed in 2011. This policy embraces the following principles supporting an enhanced regulatory framework to advance development of drugs for rare diseases:

 

  • Duchenne patients…
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Added by Pat Furlong on July 3, 2013 at 1:30pm — No Comments

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