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...]

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...]

Many articles on Spinal Muscular Atrophy…..What do they Mean?

It seems that every day, there is an unending supply of new articles posted on the web regarding Spinal Muscular Atrophy. Some articles are great...........some are horrendous...some posts seem to be unintelligible while many seem to contradict one another. While some still are very well written, but they may only be a partial view of a program which could lead to misinterpretation of the data. We try to stay as up to date on the research as possible.........Our little secret......yup we are … [Read more...]

Fundraising for Spinal Muscular Atrophy and other diseases

In diseases that are as devastating as Spinal Muscular Atrophy and others, fundraising can quickly become the priority for those who are  newly diagnosed. In what has become a highly competitive arena, new tools are created to attract new donors. At conferences across the country specific sessions are held to teach families how to become more effective in their fundraising ventures. These are all very important components of any disease community. These programs help to advance research and … [Read more...]

The Path Forward

There is a program here that has the potential to stop the progression of the disease in more advanced subjects and actually cure the disease in newly diagnosed. Wouldn't it be nice IF another family never had to hold their child in their arms and watch them fade away.............IF another family never had to live every day wondering IF today is the last day for their child. Imagine IF we as parents never had to watch our kids progress in this disease, IF we did not have to watch as our child … [Read more...]

Stem Cell Research and Motor Neurons

It was important to us to do a blog on the current state of Stem Cell Research. We have witnessed the disappointment of many in the community based on the current "Clinical Hold" that has been put on the SMA clinical trial. It is important to point out The Sophia's Cure Foundation has supported and will continue to support Stem Cell Research. We believe Motor Neuron Research is part of the big picture in regards to Spinal Muscular Atrophy. Eventually Motor Neurons will have to be replaced to … [Read more...]

Gene Therapy and Initial FDA guidelines

We feel a responsibility to keep our donors informed on the programs we have funded and continue to fund. It is the critical funding that you provide that enables The Sophia's Cure Foundation the ability to support these programs. Recently there has been some confusion as to what is necessary to advance Gene Therapy  from the lab to the patient. We have released a note recently to update the SMA community: Based upon initial talks with the FDA, the FDA believed the proof-of concept studies … [Read more...]

Gene Therapy for Spinal Muscular Atrophy UPDATE

One of the Programs we have funded and are excited about is the SMA Gene Therapy program using the AAV9  Vector (Intravascular Delivery). The Sophia's Cure Foundation has great faith in the very promising work being done at Nationwide Children's Hospital and Ohio State University. The Team consisting of Lead Researcher Dr.Kaspar , Dr. Burgess, Dr. Kissel as well as Dr Mendell is second to none. The experience of Dr. Jerry Mendell working with the FDA  in advancing programs from Proof of Concept … [Read more...]

Spinal Muscular Atrophy Mathematics-A Novel Theory

Understanding a disease as complex as Spinal Muscular Atrophy at times can become overwhelming. SMA parents contribute the majority of their  time during the day caring for their children. The research side of SMA has become my passion. So how can an SMA dad who is a construction worker by trade really understand research? Well throughout my life there were always two subjects I loved........Math and Science. I chose to become a construction worker because it was a much better living than what … [Read more...]

SMA Gene Therapy

As you all know most of our fundraising lately has been going to the amazing Gene Therapy program for SMA coming out of OSU/Nationwides childrens hospital.....we were so honored when one of the researchers, Dr. Brian Kasper asked to meet our family and especially Sophia. Dr. Kasper was in NY last week for a conference at Columbia and was so generous to spend some time with us on thursday. He was also very gracious to agree to do a video recorded Q/A session.....we asked the SMA community to … [Read more...]

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