The first question that is often asked by newly diagnosed families is when there will be a cure for Dravet syndrome. Rare diseases face common research challenges and Dravet syndrome is no exception. With a small patient population, research funding is typically non-existent and patient data is limited, making research difficult and financially unappealing to most researchers and biopharmaceutical companies. There are little to no outside funding mechanisms for rare disease research, so this heavy burden falls to caregivers to engage their community and raise money for these efforts, that in turn will offer a better quality of life for their loved one and family.Â
Our founders quickly recognized the importance of investing in scientists and clinicians who could move the needle on research. Our patient community has made significant progress with DSF working as a convener, funder, and unified voice of the patient. These efforts have provided the development of a research infrastructure through seed grants that fund basic science and clinical research to help us better understand Dravet syndrome; an annual Research Roundtable that has allowed us to share progress in the field as well as establish a multi-year research strategy; development of treatment guidelines that assure earlier diagnosis and appropriate care; educational and engagement opportunities for patient families; and continuing medical education programs for clinicians. The significant advancements in the field of Dravet syndrome we have seen since the inception of DSF in 2009 makes us confident that this approach is working, with three new FDA approved medications since 2018 and nine clinical trials currently underway, including treatments that offer the potential to not only control seizures, but may also offer disease reversal.
As part of Dravet Syndrome Awareness Month, we want to acknowledge the researchers whose life-changing work has been funded by our community through DSF. Our tagline acknowledges that progress is made possible through research, but that it all begins with community. Our community is strengthened by the inclusion of a growing number of researchers dedicated to finding answers that can impact the lives of patients and families.
Please watch our social media channels this month and next to see photos of the various labs and researchers we have funded and how these research grants have made an impact on their research efforts. And please consider ways you can get involved with DSF to join us in moving progress forward.
Community. Research. Progress.
