It is exactly what we were hoping for. Exactly what we dreamed about long ago. Big Pharma in the form of GSK has made a deal with Prosensa. PPMD and many other organizations have provided seed funding… funding in small bits to grow the work at each step along the way with the understanding that success would attract money and partnerships. The strategy worked!

Today is monumental for Duchenne.

Some months ago, I presented at BIO. Rare Disease was a focus of one of the talks. The gist of the discussion was that there would be significantly less interest and investment for common conditions. Who needs a better statin drug for cholesterol? The panel agreed, the focus will be rare disease for several reasons:

1. high unmet medical need
2. successful partnerships with advocacy (disease specific groups)
3. advocacy efforts are essential to seed projects and break down barriers
4. Genzyme and others have demonstrated investment with return in rare disease.

It is our day. PTC and Genzyme have partnered on Ataluren/PTC124. GSK and Prosensa partnering on exon skipping. And there will be more.

Thankfully, the sun is shining on Duchenne.

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Comment by Julie Hathaway on October 28, 2009 at 1:17pm
I am so thankful for you, Pat. "Now faith is being sure of what we hope for and certain of what we do not see." Hebrews 11:1.
Comment by Joanne Haswell on October 28, 2009 at 8:47am
I am in tears----Thank you God----Thank you Pat for all you do for our Duchenne/Becker families. This brings us so much hope. Lukey's grandma
Comment by Perlita & Gordy Hains on October 20, 2009 at 9:36pm
May God continue to Bless you Pat and your valiant efforts for our sons - my tears are flowing with joy and happiness - no more sorrow
Comment by Ruchika Batra on October 18, 2009 at 9:43am
Hi Pat..... it actually feels like we are indeed approaching the end of the tunner after having dreamt of it for so long.... Thanks for continuously striving for day in and day out for our sons....
Comment by Wyatt's Mommy, Melissa on October 15, 2009 at 1:18am
Thanks Pat, for continuing to fight for our boys!
Comment by Jacobs Mommom on October 14, 2009 at 1:57pm
I have chills just thinking about what this means! It is really possible that this generation of boys just might reap the benefits of these treatments!!!!!!!!!!
Comment by Ian Anthony Griffiths on October 14, 2009 at 1:42pm
Fantastic news, let the flood gates open!
Comment by JUAN PEDRO ARBULU on October 14, 2009 at 12:04am
I am feeling as if GOD has his hands on this!!!!
Thank you so much for fighting all these years Pat!!, this is your effort and we thank you so much.
Comment by Donna Taylor on October 13, 2009 at 11:30pm
Pat, we could not have come this far without you, our fearless leader. Thank you for never giving up.
Comment by cheryl cliff on October 13, 2009 at 7:29pm
The sun is shining and there is a rescue ship on the horizon. Our next treatment isn't coming in on the slow boat from China anymore (no disrespect to our Chinese friends intended). Our ship is one of those really big cruise liners from GSK. Looks first class to me!

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