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July 2012 Blog Posts (15)


Staff
CALLING ALL CALIFORNIA RESIDENTS!

A team of UCLA scientists, clinicians and clinical trialists led by Drs. Stan Nelson and Carrie Miceli have teamed with AVI and Dr. Craig McDonald to bring a novel combination therapy forward for use in DMD and sent a proposal to the California Institute of Regenerative Medicine (CIRM). It involves the UCLA discovery of an already FDA approved drug to boost exon skipping activity when used in combination with anti-sense oligo-nucleotides for Duchenne. Duchenne patient skin cells…

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Added by Ryan Fischer on July 23, 2012 at 5:00pm — 2 Comments


Staff
Transforming Duchenne Care Summary

"How can parents and patients be assured they are receiving the best care and treatment and what are the components of optimal care?”

 

PPMD gathered clinical providers, hospital administrators, industry, government, and parents together for a 2-day pre-conference meeting in order to address these critical issues. In the United States and around the world, there is considerable variability in care. Parents often feel it is…

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Added by Kathi Kinnett on July 17, 2012 at 3:41pm — 1 Comment


Staff
July Note: The Theater of Duchenne

I think at this point we all get that ending Duchenne is going to take a collaboration of efforts. Treatments will come from different researchers, produced by different pharmaceutical companies, and supported by different foundations. Ending Duchenne will be an ensemble piece, a collection of great…

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Added by Pat Furlong on July 17, 2012 at 3:30pm — No Comments


Staff
My Voice: Jack and Nolan Willis, 10 years old

The clinical trial process is complicated and confusing. At this year’s Annual Connect Conference, PPMD wanted to shed light on what the experience was like for parents, clinicians, industry, and…

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Added by Will Nolan on July 17, 2012 at 3:30pm — No Comments


Staff
Renaissance Thinking (Two Moms, Co-Blogging)

The Theater of Duchenne

At PPMD's Annual Connect Conference last month, Pat Furlong shared her essay,…

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Added by Pat Furlong on July 17, 2012 at 3:30pm — No Comments


Staff
Highlights from PPMD’s Annual Connect Conference

This year’s Annual Connect Conference in Ft. Lauderdale was incredibly informative, comprehensive, and included the leaders of the Duchenne fight. In the coming weeks, we will be…

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Added by Will Nolan on July 17, 2012 at 2:59pm — No Comments


Staff
Luca Buccella - PPMD Conference

One of the highlights of this year's Annual Connect Conference was 21-year-old Luca Buccella’s moving speech at the closing night dinner on Saturday. Son of Filippo Buccella, the President of Parent Project Italy and a friend of PPMD’s for years, Luca inspired us and made us laugh.…

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Added by PPMD on July 17, 2012 at 2:00pm — No Comments

PPMD's Participation in Clinical Trials Interview Study

Are you willing to be interviewed about your experience with clinical trials?

PPMD is conducting a study to better understand how the Duchenne and Becker community think and feel about clinical trials and potential new therapies. 

Our goal is to find new interventions to improve family wellbeing when considering or participating in clinical…

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Added by Hadar Sheffer on July 10, 2012 at 12:11pm — No Comments


Staff
Landmark FDA legislation becomes law

Major win for the Duchenne community

PPMD applauds the President for signing into law the Prescription Drug User Fee Act also known as The Food and Drug Administration Safety and Innovation Act. Most recently, PPMD worked with the Everylife Foundation for Rare Diseases and Genetic Alliance to organize…

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Added by Ryan Fischer on July 9, 2012 at 6:00pm — No Comments


Staff
Action Alert! Contact your Senators to help save vital Duchenne programs at the CDC

We need your voice to help save critical Duchenne related programs at the Center for Disease Control (CDC)!…



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Added by Ryan Fischer on July 9, 2012 at 12:00pm — No Comments

A Rollar Coaster

The trip to Six Flags was a rollar coaster of highs and lows indeed.   He was elated to go.  We made a pact to try the rides he felt comfortable with doing.  Ones that would not load his spine to worsen compression fractures from the osteopenia and osteoporosis.   We thought we had a plan.   Lot's of big "football" type boys and ride workers helped me load him into some rides. 

We were into about the seventh ride when disaster struck.   When we were lifting him into the Scrambler, the…

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Added by Jeanette Vaughan on July 8, 2012 at 9:58am — No Comments

small efforts make miracles

The heat soared to record temps of 100 degrees.  I stood on my shoreline looking out towards the small island. The lake seemed  void of any life.  Not even the ducks were out swimming now.  The water felt  almost  as warm as a drawn bath and the soft sand beneath me squished in be tween my toes.  Standing in the water felt good and somewhat cooler than the  thick July air.  I wanted desperately to enjoy some outdoor time with my sons, but the weather forecast for the next few days showed no…

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Added by Rita Felling on July 5, 2012 at 7:02pm — No Comments

What is my motivation to write?

Image One of the things that some writer's struggle with is writer's block.   I guess, maybe I am one of the lucky ones, but for me that just doesn't seem to be an issue.   I believe that some writer's block is caused by pressure.    When you feel under pressure or a dealine to produce something, anxiety is produced.   Anxiety can make the brain hyperactive, and synapse connections are definitely affected.  In fact, sometimes anxiety is so powerful, it blocks some connections…

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Added by Jeanette Vaughan on July 4, 2012 at 12:29pm — No Comments

loading

Slowly I backed  my van  toward the single  cement step leading up to my front door.  This appeared  to be the easiest way for me to load Josiah and Cody's motorized wheelchairs into the rear end of my navy blue Dodge Caravan.  I knew I would still have to use our clunky awkward but dependable, steal portable suitcase ramp to drive  the chairs across on entry.   However, I felt somewhat  relaxed with the  low incline- from going  step to van, and bridging with the ramp. A welcome relief for …

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Added by Rita Felling on July 4, 2012 at 2:08am — 1 Comment


Staff
2012 Connect Conference

Guest blog by Jill Anne Castle, mother of two boys, Oliver, 10 & Anthony, 12 (living with Duchenne). Jill works for the Arizona Department of Education in the exceptional student services division as a parents advocate and is the PPMD…

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Added by PPMD on July 3, 2012 at 12:30pm — No Comments

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