How do you measure a year?….

I would like to start this post by thanking everyone who believed in our family, all of you who believed in The Sophia’s Cure Foundation and who have believed in the programs we have funded. 2011 was such an amazing year for our organization…………..but in all honesty, I was looking past 2011 all year long because I knew what was in store for our children and family members who have been touched personally by Spinal Muscular Atrophy. To be quite frank…….I have been looking past this year and counting down the days to 2012. I know that after midnight tonight……….we are just 525,600 minutes away from an incredible year for SMA.

Together all of our families have been able to do the impossible………to take a program that we all believed in and with incredible fortitude and unwavering will power nudge this program towards the finish line………What we have as the heart in our logo is symbolic for the undying LOVE we  have for our daughter Sophia. The same LOVE that every family has for their child. The LOVE that each and every family who came together as part of the 200 Campaign shared for their children. The LOVE that literally carried this program through 2011 was all made possible by the families who came together. Now we are on the doorsteps of 2012 ready to change the world.

2012 has the potential to be a breakthrough year for our disease. The Gene Therapy Program at Nationwide Children’s Hospital recently had their Pre-IND meeting with the FDA and the data was well received. This program is moving forward towards the clinic with guidance for the Safety, Toxicology and Bio-Distibution studies needed at this stage of the game prior to moving into humans. This is the closing stretch and 100% of the funding for these studies was raised by the families involved in The 200 campaign. Your efforts assured that there would be no delays for funding reasons and when the parameters of the studies were accepted, we could march forward as a community towards the clinic. There is much work still ahead but it makes for an incredibly exciting 2012 where the goal is to be in humans. We are so very thankful to each and every one of you who have made this possible. There are so many SMA warriors who contributed to this program as well as so many friends who were near to our hearts that became Angels in 2011. It was a very painful year for all of us and especially for the families who lost a child. We were extremely moved that in their memory, their families believed in this program and literally pushed it forward for all of us. Dr. Brian Kaspar was extremely grateful for everyone who took part in the 200 campaign and wanted to thank them personally.

“Thank you for the generous and amazing support over the last year to advance our systemic gene delivery that continues to show great promise.  We have moved this program forward in 2011, gaining even greater confidence in the safety and efficacy of this approach and recently spoke to the Food and Drug Administration (FDA) regarding the advancement to human clinical trials.  We had a very productive meeting with FDA outlining a path to the clinic and we are marching forward with the required studies in 2012 to get into trials.  As always, we were impressed with the thoroughness and guidance that the FDA gave us which helps the program in general.  Our group sincerely thanks everyone involved in helping move this program and we will continue to be open and honest in our pursuit to bring a potential therapy to SMA patients.” ~Dr. Brian Kaspar Ph.D

We know this journey has been hard. Many families have been doing this for 5, 10, and even 20 years. Now is the time to push harder, stronger …never losing hope….It is always darkest before the dawn……….Our hope is this year will bring the dawn of a new era for SMA and what has gotten us all here is……………………LOVE!

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