Tap Out SMA

It has been a long time since I posted........I personally have been sitting back absorbing current events in the SMA community and sometimes that leads to stagnation. As a family we continue to move on.....focusing on today.....cherishing the moments.....living in the here and now. It is extremely difficult being a parent...trying to appropriate time evenly between your children.......especially when one of them may need special attention due to a genetic mutation. Sophia's little brother … [Read more...]

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 … [Read more...]

Limited……..Only by your Imagination…….Truly Living with Spinal Muscular Atrophy….

It has really been a long time since I sat behind a laptop to write a blog entry........................... It has been an incredibly busy year for us, and with everything going on behind the scenes I rarely have the time or energy to even write about it. Six months removed from Sophia's unforgettable trip to Disney World, I couldn't fathom anything coming close to that experience. Our most recent trip aboard the Disney Wonder Cruise ship this past week was just as amazing! Sophia is a weak … [Read more...]


August is SMA Awareness month and do we have some great news to celebrate the occasion. Let the countdown begin!*** Here is a picture of hard work. This is the paper work of a program with the best pre-clinical data ever in SMA. Thousands of pages outlining results, clinical trial design and safety for Dr. Kaspar's Systemic Delivery of the AAV9/SMN Gene Therapy Program for SMA. The best part............it has officially been received by The Food and Drug Administration. They have 30 days … [Read more...]

The 12th Hour!!!!!!

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...]

Because Research Matters………..

So what's next you guys?............. As soon as we wrapped up funding on the Gene Therapy Program that was the first question asked by so many of our supporters. The day after we received many phone calls, emails and instant messages asking us where we go from here. We cannot wait to share our vision for The Sophia's Cure Foundation and how we can further the Spinal Muscular Atrophy research community..but first............. We are so excited that SCF has wrapped up such a long and … [Read more...]

A Day For Avery…….

Three short months ago, the Skeeters were blessed with the opportunity to meet baby Avery Lynn Canahuati, who at the time was 5 months old.  Avery, who was born on 11-11-11 had been diagnosed with an incurable genetic disease and her parents were informed that she would not only lose her ability to walk, stand, sit, eat, breathe and even swallow, but that she would pass away within the next 18 months. Avery had Spinal Muscular Atrophy, or SMA for short. When diagnosed, Avery’s parents Mike … [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...]

Great Beginnings…….Sophia’s Journey through Spinal Muscular Atrophy

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...]

Avery’s Bucket List continues to touch the life of everyone around the WORLD

Avery's voice continues to be heard outside of our disease community. She has connected with people and has motivated them to make a lasting change in her memory. One of those people has approached us anonymously. This donor heard Avery's voice and wanted to make a BIG difference. Initially this anonymous donor wanted to donate $11,111.11 to honor Avery's birthdate of 11/11/11. Upon reading of  the donor willing to match every dollar that comes in Avery’s name, this generous person got all fired … [Read more...]

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