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...]

UFC vs SMA Fight Night 1/31/15

UFC vs. SMA Fight Night is gearing up to be a great event!!!! Please join us at UFC Gym Long Island in New Hyde Park NY on January 31, 2015. UFC Gym Long Island is teaming up with SCF to "FIGHT SMA" !!!!  The event will be a charity event with all proceeds going to The Sophia's Cure Foundation it will consist of UFC gym staff pairing off for some BJJ superfights as well as boxing and wrestling demos. Some of the fights include: Coach Tommy vs Coach Mike P Coach NJ vs Coach Kevin Coach … [Read more...]

First child with Spinal Muscular Atrophy has been injected with AAV9 Gene Therapy..

We are proud to announce that the first child in the Gene Therapy Program was injected just over 30 days ago. Sophia's Cure Foundation was present for this monumental day. We are looking forward to more children receiving this treatment in the near future. Thank you to Nationwide and AveXis !! We were so honored to watch history in the making, 5 years of emotions came to a head at the moment we heard "its done" Vinny and I both looked at each other and burst into uncontrollable tears......tears … [Read more...]

Amazon Smile and Sophia’s Cure Foundation team up to beat SMA!!!

We are excited to partner with AMAZON SMILE....now if you shop through AMAZON SMILE 5% of your purchase will be given back to SCF to help fight SMA!!! Just click here and start SHOPPING!!! Thank you for your supporting Spinal Muscular Atrophy!! … [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...]

KASPAR LABS GENE THERAPY FOR SPINAL MUSCULAR ATROPHY IS……..

FDA APPROVED.....we are happy to be able to share this news with you. 1,631 days ago our Sophia was diagnosed with SMA Type 1.......That same day in the living room of our small home in Wantagh, NY The Sophia's Cure Foundation was born. This has been a long, hard road; and yet one of the most rewarding journeys of our lives. We never gave up hope, we never stopped believing.  Thousands and thousands of you have stood by us, with your bake sales, collection cans, Marathon runs, Walks, Duck Races, … [Read more...]

THIS IS THE MOMENT!!!!!

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...]

Santa visits Sophia……

An amazing family friend contacted Santa and told him all about Sophia. Immediately upon hearing about what a special little girl she is he requested to visit Sophia at her home!!! Sophia loves Santa....loves the stories and the decorations and just the symbolism that Santa gives .....not just presents but gives his heart and time and love!!!! Santa to Sophia is not just about getting presents.....its about showing love, taking that extra time for a hug, or another story even though you read ten … [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...]

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