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All Blog Posts Tagged 'PPMD Blog' (711)


Staff
My Voice: Francis Desmond, 13 years old

Sometimes the best way to tell your story is through pictures. We can feel what another feels by seeing things the way they see them. Francis Desmond is an 8th grader who likes to draw. He also has Duchenne muscular dystrophy. Francis and his dad, Steven Desmond, decided to turn their journey into art and have just their father/son illustrated project, A…

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Added by Will Nolan on April 5, 2012 at 2:00pm — No Comments


Staff
Getting it all done (Two Moms, Co-Blogging)

Everyone is in a race against the clock these days. Nowhere is that more the case though, than in this community. Dealing with Duchenne, on top of everything else life throws at you, can leave you feeling overwhelmed, inadequate, and exhausted. In their monthly co-blog, PPMD President Pat Furlong and Colorado FACES coordinator, Ivy Scherbarth discuss the ways we cope with this ticking clock while keeping our families happy, our son’s healthy, and our sanity in check. Read this month’s…

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Added by Pat Furlong on April 5, 2012 at 12:53pm — 4 Comments


Staff
Great fundraising ideas anyone can try!

Are you trying to come up with an idea on how to fundraise for PPMD? Or has someone told you that they want to fundraise for PPMD, but they don't know what to do? Here are some really cool fundraising ideas that people are putting together right now in the community, which we're really excited about… Hopefully they inspire you!



Dining Out

Dine…

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Added by Danielle Garrigan on April 4, 2012 at 1:30pm — No Comments


Staff
AVI Summary Announcement

This morning, AVI BioPharma released information discussing their findings thus far in their Phase IIb study of Eteplirsen.



About Eteplirsen

Eteplirsen uses AVI’s phosphorodiamidate morpholino oligomer (PMO)-based chemistry to skip exon 51 of the dystrophin…

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Added by Kathi Kinnett on April 2, 2012 at 4:00pm — 15 Comments


Staff
Take Action: Ask Your Representative to Co-Sponsor FAST Act

We are coming to you today to ask that you devote your attention and energy towards legislation that seeks to expedite the process FDA uses to review drug applications.…

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Added by Ryan Fischer on March 30, 2012 at 9:30am — No Comments


Staff
Bikram Yoga meets the Grand Canyon

Bikram yoga. Michelle had just been home talking about her Bikram practice. We talked about it a lot over the holidays. Michelle had been practicing for nearly 5 years. I wondered if I could survive 90 minutes in 105 degree heat. I had a range of readily available excuses; the easiest one was that there was no Bikram in Cincinnati, Ohio. And my unspoken reason was that I hesitated because I…

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Added by Pat Furlong on March 28, 2012 at 11:00am — No Comments


Staff
Prosensa’s Clinical Development Plan Update – March 2012

In parallel to the on-going development of the lead compound PRO051/GSK2402968, which is being developed by GSK with an active involvement of Prosensa, there are five more compounds for Duchenne in Prosensa’s pipeline.  Below you can find an update about the current state of development of these compounds.



Prosensa is currently conducting a clinical study to investigate the effect of PRO044 in boys with Duchenne muscular dystrophy, who have a dystrophin gene mutation amenable to…

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Added by PPMD on March 26, 2012 at 9:41am — 10 Comments


Staff
Steroids: A Certain Can of Worms (A Complicated State of Affairs)

Steroids. What regimen, what dose, for what individual? When to start? When to stop? A complicated question to be sure.

 

Steroids are a complicated story and have clearly changed the natural history of Duchenne. We know they work for some, for most in fact, but not all. We know they increase strength and prolong ambulation, and statistics back this up. Predicting ambulation is prolonged on average for 2+ years. But this is not the end of the story. Genetic modifiers have been…

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Added by Pat Furlong on March 20, 2012 at 2:00pm — 7 Comments


Staff
Important Webinar: AVI Clinical Trial Update

AVI Clinical Trial Update

Parent Project Muscular Dystrophy presents an AVI webinar on Wednesday, March 21 at 1pm eastern. The webinar will cover a pre-data update on AVI BioPharma's Phase IIb study evaluating eteplirsen for the treatment of Duchenne patients with mutations amenable to exon-51 skipping, and will be lead by Chris Garabedian, the CEO of AVI…

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Added by PPMD on March 19, 2012 at 2:30pm — No Comments


Staff
Our View

So many times we view the world from the dark side, the part where we worry our sons (and daughters) may never see x or y, may never have this or that opportunity. I recall Kevin Smith standing up and telling us how he promised Zach there would be no limits on what he wanted to do or see, that if he wanted to see the top of a mountain, Kevin would put him on his back and show him the view. I think any of us would do that for our child or for our friend.

 …

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Added by Pat Furlong on March 14, 2012 at 4:30pm — No Comments


Staff
My Voice: Ricky Tsang, 30 years old

Our Featured Voice for March 2012 is 30-year-old, Canadian author, Ricky Tsang. If you are part of the Facebook world, you probably are friends with Ricky or have at least seen his name around. He is the author of the recently released, …

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Added by Will Nolan on March 13, 2012 at 4:30pm — No Comments


Staff
Rare Disease Leaders’ Forum 2012

This is my first visit to San Francisco, the city by the bay, home of the Golden Gate Bridge and Tony Bennett’s song “I Left My Heart in San Francisco,” a song from my younger days and one that, even today, sometimes gets in my head and repeats and repeats.



Some time back I was invited to…

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


Staff
Volunteers needed to Dine Out for Duchenne!

Do you love to go out to eat? Do you want to help fundraise, but don't have the time? Then this is the event for you!! You can participate whenever you visit a new restaurant without doing extra work! It's fast and easy!! 

 

Dine Out for Duchenne is a week-long, online event where people can…

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Added by Danielle Garrigan on March 12, 2012 at 4:30pm — No Comments


Staff
Co-Blog: Gravity

PPMD President Pat Furlong and Colorado FACES Coordinator Ivy Scherbarth discuss the bravery of their sons, and all young men fighting Duchenne. Please, share your thoughts after reading this month’s co-blog.



Strength

by Pat Furlong

Weakness. A familiar word in the context of…

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Added by Pat Furlong on March 8, 2012 at 4:30pm — 3 Comments


Staff
Reminder: Register Today for 2012 West Coast Connect Meeting

By now we hope you have heard that Parent Project Muscular Dystrophy will be hosting our first West Coast Connect Meeting, April 13-15, 2012 in San Diego, CA at the…

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


Staff
Vote for Duchenne in the 2012 Neuro Film Festival!

The trailer for "Duchenne With A Future: The Power To Live," a documentary about the success factors of living an adult life with Duchenne, has been entered into the American Academy of Neurology's 2012 Neuro Film Festival! By enrolling we hope to promote research and raise Duchenne awareness.…



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Added by PPMD on March 6, 2012 at 7:00pm — 1 Comment


Staff
Everyone Gives

Everyone gives something to somebody for something. Whether through acts of kindness, services, or hard-earned money, everyone has the opportunity to make the world a better place. No energy required. 

 

Literally from the comfort of our couch, we are able to reach across the world to support others in need. 250,000 boys/young men have Duchenne. They live everywhere,…

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Added by Pat Furlong on February 22, 2012 at 3:00pm — No Comments


Staff
PPMD Announces Board Approved FDA Policy

As you know, ensuring appropriate federal funding for Duchenne research has been a cornerstone of PPMD’s advocacy agenda over the past decade. Our persistence – your persistent and collective voice - has paid off with over $250 million directed to Duchenne research.

 

Today we now find ourselves at a pivotal moment with multiple potential therapies for Duchenne in various stages of the development process. We must now, once again, look forward to what is next for Duchenne.…

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Added by PPMD on February 21, 2012 at 10:00am — No Comments


Staff
PPMD FDA Policy and PDUFA

I don’t know about you, but when I am in DC or receive government related information, I am always about 3 acronyms behind in the conversation.  PDUFA – this is one acronym you want to put in your memory bank for 2012.  PDUFA – Prescription Drug User Fee Act.

 

This legislation is all about FDA, about what biotech’s/pharmaceuticals have to pay for the process, about rare disorders and incentives to develop drugs for rare conditions, and about the process of…

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Added by Pat Furlong on February 20, 2012 at 10:30am — 5 Comments


Staff
Go West! Join PPMD in a variety of West Coast events!

We are excited to announce a 2012 calendar full of events on the West Coast!

 

West Coast Connect Meeting

First, PPMD is excited to host our first West Coast Connect Meeting, April 13-15, 2012 in San Diego, CA. This will be a great opportunity to…

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

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