“Your voice matters. You are the expert in your field. Educating the leaders who make critical policy changes about what you are doing is the only way change is made.”
—Monica Weldon, President/CEO/Founder,
Bridge the Gap – SYNGAP Education and Research Foundation
When Monica Weldon's son, Beckett, was four months old, she noticed he was not meeting the same milestones as his twin sister. Monica then began a journey to find answers to help her son. In November of 2012, at age 4, Beckett was diagnosed with the gene mutation SYNGAP1 (6p21.3), linked to autism, epilepsy, and intellectual disability. When Monica started to blog about Beckett's progress, she began to build a community of parents and caregivers that are now an active support group. She retired in 2016 after 23 years in education teaching secondary science, to start a new mission.
She founded Bridge the Gap – SYNGAP Education and Research Foundation, of which she is president and CEO. The Foundation aims to support these "SYNGAP1 families" by raising awareness and accelerating therapeutic research. Weldon is the primary investigator on the SYNGAP1 (MRD5) Registry and Natural History Study and an author of several high-impact articles in neurology publications. She is an author, public speaker, consultant on rare disease business strategies, and advocate for rare disease legislation at both the federal and state levels. Monica also authored a book about Beckett's diagnostic journey, called Slow Moving Stream: My Special Boy. She was a 2015 and 2016 Global Genes RARE Champion of Hope advocacy award nominee for her work in the rare disease community and WEGO Health Awards nominee in 2015, 2017, 2018 and 2019.
Weldon is a speaker in the upcoming Advocacy in Action Interactive Lunch Workshop at ANA2019. We spoke with her about the importance of advocacy, and how neurologists and neuroscientists can help.
Q. Why is advocacy for neurological disease research important to you?
The patient voice is one of the most critical aspects of making changes that will benefit patients. Advocacy is key to raising awareness, educating the public, experts, and policymakers who will help move forward the need for treatments in disease.
Q. Are you involved in other advocacy efforts aside from Bridge the Gap? If so, what are they?
I am an advisory member on the policy committee of the American Brain Coalition. We advocate for funding for neurological conditions, asking for federal funding for the NIH’s BRAIN Initiative and other government programs that support neurological research. I'm also a member of the Community Congress Policy Committee with the EveryLife Foundation. Currently, we are working on an economic burden study for rare diseases. Our committee also focuses on advocating for the OPEN Act, which will help create incentives for the pharmaceutical industry to invest in rare disease drug development.I am an advisory member on the policy committee of the American Brain Coalition. We advocate for funding for neurological conditions, asking for federal funding for the NIH’s BRAIN Initiative and other government programs that support neurological research. I'm also a member of the Community Congress Policy Committee with the EveryLife Foundation. Currently, we are working on an economic burden study for rare diseases. Our committee also focuses on advocating for the OPEN Act, which will help create incentives for the pharmaceutical industry to invest in rare disease drug development.
Q. As a patient advocate, why do you think it is beneficial to have neurologists and neuroscientists join in with lobbying and advocacy efforts? How would you recommend they start?
It is critically important to come together on collaborations and incentives that will help drive the funding into researching diseases. Without investment in basic science research, there would be no fundamental understanding of disease to target optimal treatments.
Researchers who are members of professional associations should get involved by getting to know the policy directors of that institution. It will help them better understand current issues and policies and create opportunities to get involved at a much deeper level. Getting to know their local representatives and senators is a great place to start. Most politicians need exposure on the issues that researchers face with funding.
Q. What one or two things are most important for academics, neurologists, and neuroscientists to know about engaging in political advocacy?
Your voice matters. You are the expert in your field. Educating the leaders who make critical policy changes about what you are doing is the only way change can happen.
Q. Do you have stories of successful advocacy that you can share? What made those efforts successful?
Even though our patients do not yet have a treatment, planning for the future and getting involved can help remove future barriers. Our organization was a part of the influence that helped get the Rare Pediatric Disease Priority Review Voucher Program pushed through the House. This piece of legislation eventually was passed into law as part of the 21st Century Cures Act. We are also a proud member of the Texas Newborn Screen Task Force, which came together to create the Newborn Screening Preservation Account. This account will allow Texas health officials to maintain and expand the state's genetic disease testing program for newborns, in the hope that this will help ensure that SYNGAP1 and other conditions can be added to the required screens once treatment is found.