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 non-ambulatory boys -
outcome measures for non-ambulatory boys will be validated
Amazing interest from Biotech and pharma and partnerships- Genzyme/PTC; GSK/Prosensa
Champions in the US Congress who believe and support our goal to treat and cure Duchenne.
Amazing researchers and physicians
And a world of families working together.
Today boys with Duchenne are walking longer and living longer. New therapies to extend function are in development and in trial. In 2010 we will see trials with Sildenafil, Losartin, exon skipping, gene therapy (limb delivery) and many more. Today, we have more time with our sons. Tomorrow, in 2010 , the future will be even brighter.
Thank you for your friendship and for your support. You are changing the world of Duchenne.
Warm regards for a happy, healthy New Year.