Representing our entire research team, I am standing here humbled and with profound gratitude to express our deepest appreciation for your remarkable support of our research. Your belief in the power of biomedical science, your trust in my team, and your generous gift are truly amazing and impactful. They have played an indispensable role in advancing our SCN2A studies toward a transformative path of precision medicine.
I still vividly recall attending the 2nd SCN2A conference back in 2017, when I just started my lab. At that time, our resources were limited, and I was the only researcher in the entire lab. While I knew no one at that conference, I was warmly welcomed by so many dedicated families. Two years later, at the Seattle conference, I brought my mother, who was terminally ill, to spend her last days with me. In this big meeting with hundreds of people, my mother and I met with Leah very briefly. But several years later, Leah still remembered my mother and asked about her. It was very emotional for me. FamilieSCN2A is not only my professional home, but also a loving family for me at the personal level. I guess “families” is part of the name for a good reason.
Over the past few years, our lab has kept growing. In fact, now we have over a dozen researchers with diverse backgrounds in the lab. Some of them are here at this conference today: Jingliang, Xiaoling, and Sunny from China; Yen from Korea, Manasi from India, Morgan from Canada, and Kyle from Indiana. They are the driving force behind all the discoveries we produced in the lab. And I am very proud to state that every single one in the lab is united under one mission, which is to push into the boundaries of science to find a cure for SCN2A disorders.
This journey from me as a single PI to a dedicated team of researchers to study SCN2A was certainly not possible without substantial support from private donors like yourself and federal agencies like the NIH. But government funding does not come by easily. During our challenging time, we were immensely grateful to receive the Action Potential grant. This grant provided us with the hope and means to sustain our operations and retain our talented researchers during that difficult time. And eventually, allowing us to obtain large NIH funding with a 200-times return on investment.
Most recently, we are extremely privileged to receive the Hodgkin-Huxley Research Award, made possible by generous major donors. With the support from this award, we aim to advance n=1 translational research — an endeavor that is rarely funded by NIH or pharmaceutical companies. This substantial Award has empowered us to undertake high-risk, high-reward projects, explore novel therapeutic strategies, and pioneer cutting-edge technologies that hold enormous promise of transforming lives.
In fact, with all the generous support, our team has generated a wealth of data over the past years, resulting in seven posters at this conference. Some of the studies describe the new disease models we are building; others show off our ongoing gene therapy efforts. These posters stand as a testament to our commitment to combating SCN2A disorders day in and day out. I encourage you to check them out if you are interested.
As we express our heartfelt gratitude today, we want to assure you that your gift will continue to fuel our determination and inspire our relentless pursuit of a cure for SCN2A disorders. We are committed to charting new courses, leaving no stone unturned in our quest to find effective therapies.
We are gathered at Disneyland, the land of magic today. Here we believe dreams come true and magic is real. Talking about magic being real, there was actually an interesting saying. It goes, “Any sufficiently advanced technology is indistinguishable from magic”. I agree. With your support, we are taking the science adventure to make magic real. Together, we are shaping a future where SCN2A disorders are conquered, and the promise of health and well-being is fulfilled for all of our children.
We will fight for you until that magical future arrives.
Thank you!