Dravet Syndrome Foundation (DSF) offers programs that bring great value to the Dravet community through three primary pillars – Research; Professional Education; and Caregiver & Patient Support. Our community has done many important things that have helped to move the field forward. DSF’s grant program has awarded over $4.1M through 43 research projects. Thanks to our professional education efforts, more patients are being diagnosed earlier and receiving appropriate treatment. And DSF’s patient and caregiver programs assure that our community is educated and connected.
But, would it surprise you if I said: One of the most important things that DSF does is fundraising. Fundraising leads to awareness. Fundraising leads to research. Fundraising leads to breakthroughs. Fundraising will one day lead to a cure.
It is difficult to research rare diseases such as Dravet syndrome (DS). The research necessary to find cures requires money – money that is currently not being funneled towards rare diseases. When funds are scarce, you have to be creative. Our community couldn’t just rely on the fact that perhaps eventually governmental agencies such as the National Institutes of Health (NIH) would begin to fund more research on DS. Not when we were watching our loved ones living with DS who needed better options now.
DSF saw the potential of investing in both established researchers and young investigators who had an interest in DS. Pilot funding would allow them to gather the data they needed to apply for larger funding, while also drawing them into the field. While we are very fortunate to have generous industry partners who fund caregiver and patient initiatives, they don’t contribute to our research programs. The research DSF has funded comes exclusively from private funds.
As a parent myself, I recognize that science never moves fast enough when you want and need a cure for your child. But, you should be excited about the current state of research into Dravet syndrome. When you look back at the last 15 or so years in the field of DS, we have seen significant accomplishments:
- The discovery of an SCN1A mutation associated with DS, giving researchers a genetic marker to focus on and allowing the development of genetic testing for early diagnosis.
- Identification of hundreds of existing patients in the US previously not diagnosed, and early diagnosis of hundreds of new patients.
- Development of a North American Treatment Consensus to assure appropriate diagnosis and best care practices for patients.
- Establishment of a more accurate incidence rate than originally believed.
- Development of several mouse models, two zebrafish models, and a plethora of iPSC models.
- Identification of more than 1500 SCN1A mutations in patients, allowing for robust studies on phenotype/genotype correlations.
- Expansion of federal funding for DS/SCN1A research from about $1M in 2005 to $12M in 2016.
- Two FDA-approved medications with indication for DS, and a third that will most likely receive approval within the year.
- Interest from multiple industry partners who are exploring upregulation and/or gene therapy treatments for DS.
I understand first-hand that each of our caregivers is not only dealing with a devastating disease, but now I am asking you to find the strength and time to advocate, to raise awareness and to raise funds. While it might seem too much to add to what you do each day, the fact remains that it falls to those of us who have loved ones with DS to assure research continues to happen at the fastest pace possible.
DSF is designated a “non-profit” for a reason. Any and all revenues go back into the organization and its programs. But every bit of work that DSF does requires funding. Research, community events, and scientific meetings all require funds in order to operate. The strides forward that we have made could never have happened without the support and passion of the community that makes up DSF – including researchers, clinicians, industry partners, and most importantly, our families.
You can make a difference year-round by raising awareness, raising money, and advocating for patients living with DS. There are 3 primary ways you can help:
- Share your story – Sharing your story about how DS affects your child and your family is a powerful thing to do. People donate to causes that touch them or inspire them. It is the stories of our children that teach others about DS and puts a face to the disease. Email DSF about ways to participate.
- Host an event – One of the biggest hurdles we face is the lack of awareness of DS. Hosting an event is a great way to start a conversation about DS, while also raising funds. We understand the work involved in organizing an event of any size, so we have programs developed to make it easier for you. Our Campaign Director, Misty Ried, is available to help you every step of the way.
- Participate in an event – You can raise awareness and funds by participating in an event near you or through a virtual event – such as DSF’s Give Up Your Cup or a Facebook Birthday Fundraiser. These are easy ways to raise a few hundred dollars or more while spreading awareness of DS. The cumulative effect of these efforts is astounding, and every dollar really does add up. For example, in 2018, Facebook Birthday Fundraisers raised almost $100,000 for DSF – enough to fund two Postdoctoral Fellowships.
As Margaret Wheatley said, There is no power for change greater than a community discovering what it cares about. You already know what you care about. I encourage you to join DSF in continuing to make positive changes for our community. You can always email me with any questions on DSF and its programs, or email Misty about getting started with fundraising.