Recently we traveled to Washington D.C. to participate in the public RAC (RECOMBINANT DNA ADVISORY COMMITTEE) meeting on behalf of Dr. Kaspar's Gene Therapy for SMA....We have been supporting, funding and following Dr. Kaspar's program for just about the last 3 years. We knew this meeting would be step 1 of 3 critical steps that Gene Therapy must go through to move to clinical trials. Once we heard the date announced we quickly contacted the NIH to report that we would like to speak and present … [Read more...]

Life seems to always present parallels. The world will never forget 911.......A day when countless innocent souls were attacked by a hatred that seems unfathomable. The World Trade Center took many years to build.........men and women joined together to create what would one day become  the symbol of American prosperity. It was no easy task........but thousands of hard working union men and women each played a role and worked towards a common goal.......Every day these hard working tradesmen … [Read more...]

Somewhere through this journey.........we have been able to let go of the fear, let go of the pain and to enjoy this journey together......as a family. It is hard to pinpoint when the transitioned happened......it just did. Individually and collectively as a family things have gotten so much better than they were even a year ago. I know as a father I have been able to put so much anger, hurt and questioning to rest,..........and to begin to Love life in it's entirety again. I have been fortunate … [Read more...]

Today is a day of both overpowering grief and overpowering hope in the SMA Community. As we described in our previous blog post, beautiful Avery Lynn Canahuati, of "Avery’s Bucket List" passed away yesterday at just 5 months old. In their latest blog, the Canahuati family asked that donations be made in Avery’s honor to Sophia’s Cure Foundation to help fund Dr. Kaspar’s gene therapy program, which will be headed to human clinical trial by late 2012 or early 2013. As many of you in the … [Read more...]

Super SMA KID Cashel will be turning 16 soon, he took it upon him self to start SMA IT FORWARD......He is hoping his Facebook page will reach 1 million likes by the time he turns 16....So lets get out there and get our friends and family to make this happen. and Sharing is caring :) CLick here and like  SMA IT FORWARD.... We are pulling for you Cashel. … [Read more...]

The time has come to thank the many families and groups that have come together as part of the 200 commitments for SMA. Your efforts have pushed a program forward at a pace that has rarely been seen in our disease community. The community should be extremely proud of what they have been able to accomplish. Last year at a research conference I remember a representative of a panel state that a smaller organization could never fund any of these programs to the clinic. With your support.........we … [Read more...]

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 … [Read more...]