Thank you for believing in us and SOPHIA!!!

We are very proud to announce enrollment for the Gene Therapy Clinical Trial is now OPEN.
Phase I Clinical Trial of Systemic SMN Gene Therapy For Spinal Muscular Atrophy To Begin at Nationwide Children’s Hospital in May. The purpose of this trial is to evaluate safety and efficacy of intravenous delivery of self-complementary scAAV9.CB.SMN as a treatment of Spinal Muscular Atrophy type 1 (SMN1).

This was a program Sophia’s Cure Foundation lead the way in with a multi-million dollar investment. We made our first donation in 2010 and continued our financial support every year since. Our proudest accomplishment was funding all of the product that will treat the SMA babies for the very first time. We are very proud to have spearheaded the effort to insure that this program was fully funded. There were many doubts that a Mom and Pop 100% volunteer non-profit could support a multi-million dollar program through the clinical trial process. The message started here but traveled throughout the world. We could not have done it without your help..each and every donation, and fundraiser you did made a difference. We are very thankful for the enormous support from Avery’s Bucket List. The Little Aviator Avery was monumental in pushing this program forward. She moved the world and made sure this would get to trial. Every parent involved in the 200 campaign helped push this forward. Together we brought the Giant to It’s Knees. We did this together and against all odds. Thank you for believing in us and in SOPHIA!!! We love you all !!!!

Click Here for information on enrolling into the Clinical Trial.


  1. Dear Vincent, Catherine, Sophia and Jackson.
    Today you should all be very proud of this outstanding accomplishment. Your perseverance and desire reminds me of the story of David versus Goliath.
    Your family has worked tirelessly over the past 4 years to make this program a reality. Everyone involved in any way with SMA, owes your organization a debt of gratitude.
    I join you, your donors and families today in celebrating a victory today.
    The hard work is still ahead of us and I look forward to your continued involvement as we all move the program forward.

    Congratulations! From your friends at AveXis

    • Dear Dear Vincent, Catherine, Sophia and Jackson.. and you NEW friends at AveXIs 😉
      thanks you so much for making this happen.. the entire SMA community is crossing their fingers 😉 We hope that moving forward more SMA Types and Older Adults will also be able to participate in this History Making Incredible treatment that is finally moving forward!!!!! Thank youThank youThank youThank youThank youThank youThank you
      (Ambulatory Type 3, 40yrs)

  2. I’m in tears! Finally…

  3. Dear Vinny and Catherine,
    This news is extraordinary!!!! The two of you have taken on a giant! You didn’t let the system beat you down.
    Vinney, I am proud to know you and Catherine, I know we will meet some day. Before Sophia, I had never heard of SMA. You guys put it on the map. Sophia, your life has literally changed the world and I know Jackson is going to grow up to be a better person because you are his sister.

    God Bless you all. And all the moms, dads, and their children who have been fighting SMA.

    Elizabeth Barton

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