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...]

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...]

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...]

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...]

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...]

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 … [Read more...]

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...]

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...]

Nationwide Children's Hospital receives $250,000 grant for SMA research Brian Kaspar, PhD, principal investigator in the Center for Gene Therapy at The Research Institute at Nationwide Children's Hospital, along with a team of Spinal Muscular Atrophy (SMA) researchers and clinicians, recently received a $250,000 grant for SMA research and clinic development from Sophia's Cure Foundation via the Pepsi Refresh Project. Spinal Muscular Atrophy (SMA) is a group of inherited debilitating … [Read more...]

Everyone this is it! This is what we have been funding. He has made it to Esquire! We have played a huge part in speeding up this trial. Every dollar we have raised has been going to this trial. The more we raise the faster it starts. Thank you for your continued support. Hopefully very soon we can start saving babies! EsquireMagazineArticle … [Read more...]