I remember when Sophia was first diagnosed and Vinny and I started meeting other SMA families online …we would look at all the photos of the beautiful children. I will never forget when we first came across Aiden’s photos……his big beautiful smile was so infectious it just made you want to smile. It was hard to look away!!!!!! You could see the happiness radiate from him, you could just tell all the love and happiness his mommy and daddy brought him just poured out from him!!!! We were heartbroken when Aiden lost his battle to SMA, nowhere near what his mom and dad must feel. But if I know one thing, that beautiful boys smile is branded in our hearts and mind,  I can still see it……..nothing and nobody can take that away….We want to thank all the generous donors who donated in memory of Aiden Bundy. All funds raised in Aidens name will be going to Dr. Kaspar’s Gene Therapy Research for SMA, requested by his mom and dad, in hopes that in the near future SMA will no longer be a terminal diagnosis.

Dear Vincent

Some very dear friends of my wife and mine recently lost their daughter to SMA. Her name was Sophie Kathleen TerHorst. She was a beautiful little girl who brought tons of joy to the world in her short nine months with her family and friends.

Sophie’s fight with SMA shed light on the character of her parents, Mike and Liz, too. They were determined to give their daughter  the highest quality  of life while alive and handled this terribly tragic circumstance heroically. Organizations like yours helped Mike and Liz make the most of the short time they had with Sophie.

Because the TerHorst’s are such good friends and my wife and I were so impressed with their handling of their daughter’s condition we offered to make a donation of $2,500 to the organization of their choosing on Sophie’s behalf through The Lutsey Family Foundation.

Mike and Liz decided to donate the funds to your organization! On behalf of Sophie kathleen TerHorst, please accept the enclosed check of $2,500 for The Sophia’s Cure Foundation.

Sincerely,

Andrew Lutsey

We are so grateful and humbled by the generous gesture from baby Sophie’s family, all funds will go to Dr. Kaspar’s Gene Therapy program. Another tiny hand making a big difference to end 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!

Soon after we heard this news Sophia became very ill with a UTI infection……after hearing about Diana’s positive experience at Winthrop we packed Sophia up and went straight to Winthrop ER……we entered the ER and told them about Sophia’s High fever and Heart Rate and that she had SMA, and needed to be kept isolated….No questions asked they immediately brought us to a private ER room to be evaluated. Soon after Sophia was admitted to the PICU (pediatric Intensive Care Unit) The PICU was in a private end of the building and every patient had a private room. Sophia received excellent care and during our stay we met with the Chief Pulmonologist and the Chief of Intensive Pediatric Care. We told them that the reason we brought Sophia in was because of Baby Diana’s positive experience, they all knew Diana very well, and commented on how Denise and Marcelo were amazing proactive parents. We spoke more about Dr. Schroth and they were all so willing to meet her and learn about SMA respiratory  care.

Soon after we were back at Winthrop for Sophia’s Trach surgery, and through our two week stay the Pulmonary Dept was constantly working with Dr. Schroth to help give Sophia the best care.

Recently we were overjoyed to here that Dr. Schroth was making a two day trip to NY, (as if she is not busy enough) to meet and Teach the Winthrop Pulmonary Dept about SMA Protocol. We have heard that this experience has been extremely positive and everyone was eager to learn. Dr. Schroth is one of the best in SMA Respiratory care and we are so excited that Winthrop and Dr. Schroth were able to make this important meeting happen.

So baby Diana even though your stay on this earth was brief, you have made a huge difference, none of this would have happened if you and your mom and dad had not paved the way. Thank you for finally helping us all have a Long Island hospital on board with SMA Protocol!!!!

1) There must not be an IPAD in the HOME

2) For SMA Kids/adults only

Soooooo how to enter:

1) Comment on this blog post below please include the following…

a) Full name of SMA kid/adult

b) let us know how the Ipad will improve their day to day life.

Thats it!!! Good Luck to everyone….. entries will be taken up until midnight saturday Nov 5. The winner will be drawn on Sunday morning!!!!