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Featured Blog Posts – December 2009 Archive (5)

Staff Happy New Year

Dear Friends,

It is the last day of 2009, the end of the decade. It seemed every conversation started with 'in this economy' and we all worried what those words might mean for progress in Duchenne, for buying time. As a community, we held tight to each other, to our sons and to our hope. We end on a high point:

Ataluren pivotal trial

Validated outcome measures for ambulatory boys

Antisense oligonucleotides in systemic trial (Prosensa and Avi)

Alaluren trial for… Continue

Added by Pat Furlong on December 31, 2009 at 3:29pm — 5 Comments

Staff The "C" - Part 1

My friend Mela says I signed up for this a long time ago, several lifetimes perhaps, in a book I don’t recall and in a place I don’t remember. She believes in reincarnation, that we come back to learn things, important things, necessary things. If so, I would be interested in making the argument that we should at least get to remember what we learned in previous lives. It is only fair. I cannot argue for or against reincarnation. I simply don’t know. I can see the utility in recycling souls,… Continue

Added by Pat Furlong on December 16, 2009 at 2:53pm — 15 Comments

Staff Welcome news!

Ataluren (PTC124®) in Nonambulatory Patients With Nonsense-Mutation-Mediated Duchenne/Becker Muscular Dystrophy (nmDMD/BMD)

I realize it is so difficult to see trials targeting specific mutations and feel like you are on the outside looking in, wondering when it will be your son’s turn. Prosensa and AVI’s trials are moving forward as well. More trials are in the planning stages – Sildenafil, Losartin, IGF-1 and others. Still some of our boys do not fit the criteria, either… Continue

Added by Pat Furlong on December 11, 2009 at 2:59pm — 6 Comments

Staff Care Considerations Available

Care Considerations – to be published in Lancet – is available now, online . These Care Considerations set the stage for all boys everywhere to receive optimal care. Families now have access to this document as well as an abbreviated, family-friendly version currently in development. This is your guide and your doctor's guide to good care. Your questions, your concerns are… Continue

Added by Pat Furlong on December 7, 2009 at 12:34pm — 6 Comments

Staff Josh Winheld - In memory

Saywatanayo (Shawshank Redemption)

To my friend Josh,

I’m sure heaven is a brighter place today, but our world feels pretty empty. We did not have time to say goodbye. Or time to tell you all of the things that come to mind today or how thankful we were to know you, to have you in our lives, to learn from you, to see you smile.

You had learned all of the lessons life had to teach. You shared your knowledge, spread your joy and extended your hand to all of us.… Continue

Added by Pat Furlong on December 6, 2009 at 6:54pm — 7 Comments