Rare Leader: Trish Flanagan, co-founder and president, YBRP
May 20, 2021
Name: Trish Flanagan
Title: Co-founder, president, and director
Organization: YBRP, Inc. (The Yellow Brick Road Project)
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Disease focus: Heterogeneous nuclear ribonucleoprotein H2 (HNRNPH2) is a gene in humans. HNRNPH2 genetic mutations result in a neurodevelopmental phenotype that includes developmental delay, intellectual disability, hypotonia, and seizures, among other characteristics. This was first identified in “Variants in HNRNPH2 on the X chromosome are associated with a neurodevelopmental disorder in females” by Jennifer Bain and Wendy Chung, published in the American Journal of Human Genetics in September 2016. In 2016, six girls were identified with variants in the HNRNPH2 gene in a study by Jennifer Bain, pediatric neurologist at Columbia University. She found that each individual had developmental delay or intellectual disability, as well as atypical muscle tone. Many of the girls also experienced seizures and conditions such as anxiety and autism spectrum disorder (ASD). They discovered variants in HNRNPH2 also seemed to affect other organ systems in the body aside from the brain. For instance, patients with variations in HNRNPH2 also had problems with growth, gastroesophageal reflux disorder, scoliosis and other skeletal problems, heart problems, and atypical facial features.
Headquarters: New Rochelle, New York
How did you become involved in rare disease: In 2016, our diagnostic odyssey for my daughter’s global developmental delay ended with the results of whole exome sequencing. In 2017, four families met with a neurologist and geneticist out of Columbia University in New York and we decided to put on the team shirt and chase down a cure.
Previous career: I still haven’t given up my early childhood teaching career. Life is a true juggling act.
Education: B.S. human development and family studies with a focus in early childhood and an M.S. in early childhood education
Organization’s mandate: To connect families, raise awareness, and drive research into HNRNPH2 forward leading to treatments and a cure for HNRNPH2.
Organization’s strategy: As the name implies, we know this endeavor will be a journey. We know that as we grow, our momentum will too. We are in this together, and will take each step and make each decision based on whether it points us in the direction and/or gets us closer to a treatment and cure for the children we love.
Funding strategy: As a small grass roots effort, we’ve depended on small grants but mostly the efforts of families touched by the condition picking up the torch and activating their networks to help the YBRP’s work. There are three high fundraising holidays of HNRNPH2: Rare Disease Day, H2 Awareness Week in late July, and Giving Tuesday.
What’s changing at your organization in the next year: We will be launching a YBRP data initiative with COMBINEDBrain and the Across Matrix Rare Disease Platform. It’s really essential for us to broaden our bandwidth to further our reach, begin work towards a standard of care document, and of course fundraise, fundraise, fundraise. We need to find more of the tribe that we know are out there and are planning a campaign called Diagnosis Matters through our Outreach and Action Committee to help to make genetic testing more accessible and affordable to all. We are eager to learn the results of some preclinical trials going on this year that we know will lead to clinical trials. Identifying meaningful endpoints and outcome measures for those clinical trials is also high on our long list of plans for the next year.
Management Style: Visionary—I want to keep our organization’s vision at the heart of every decision we make and try to keep the end goal of a treatment and cure front and center as members of the organization are given latitude to contribute, make decisions, and do the work that they are good at doing.
Management philosophy: Democratic—I really want and consider everyone’s input. We make decisions together.
Guiding principles for running an effective organization: Action is expected. Participate, collaborate, and create. Wear many hats to fill in gaps as for the time being, we are stretched a bit thin.
Best way to keep your organization relevant: Keep finding more of the tribe. Our voice will be louder and louder as our numbers grow.
Why people like working with you: I think my hope and deep well of positivity and determination to have an impact inspires. I express gratitude and genuinely see others’ skill sets, particularly those that are unlike my own, as such assets and don’t hide how important those skills are to the success of the organization. I never pretend to know it all nor do I shy away from a debate. I have a growth mindset, so I’m an active and I’d say good listener, but will express my position after careful consideration. I work hard for everyone to feel on the same page and on board with decisions that are being made.
Mentor: Monica Coenraads [Rett Syndrome Research Trust] and Matt Wilsey [Grace Science Foundation] both gave early advice that shaped how we decided to launch and advance the project and most recently, TerryJo Bichelle [COMBINEDBrain] absolutely inspires me.
On the Job
What inspires you: I love meeting and welcoming new families and orienting them to the diagnosis and the community that will support them. These are my people and I am so happy to connect with them.
What makes you hopeful: I’ve always had a very positive outlook, even before this despicable diagnosis came for my daughter, Morgan. In many ways, stars seem to be aligning for the HNRNPH2 community and for such a young organization, the YBRP is doing remarkably well. Clinical trials aren’t far off and this is exciting and hopeful to be on the brink of reaching.
Best organization decision: Expanding our board of directors and establishing committees comprised of other HNRNPH2 parents. What a difference it has made to have more hands on deck. They’re folks that are just as passionate about the mission as I am.
Hardest lesson learned: To know the treatment isn’t or wasn’t in time to help those patients who experience some of the most devastating effects of this disorder. It’s a very hard pill to swallow.
Toughest organization decision: Funding our first research project. Toughest, but most exciting all in one.
Biggest missed opportunity: We were very hopeful that a partnership we’d established that made use of facial recognition technology would lead to the identification of more people with HNRNPH2 mutations. For various reasons, that has not been the case. Quite a disappointment.
Like best about the job: Partnerships and relationships forged for this greater good of helping individuals with rare diseases.
Like least about the job: There are just not enough hours in the day.
Pet peeve: Tardiness
First choice for a new career: Writer or dog trainer
Personal Taste: I’m low maintenance and happiest in good company.
Most influential book: The Universe Has Your Back by Gabrielle Bernstein
Favorite movie: Terms of Endearment
Favorite music: Easy listening—James Taylor, Adele
Favorite food: Sushi / Sashimi
Guilty pleasure: Haagen-Dazs Coffee Chip Ice Cream
Favorite way to spend free time: On a golf course with my best friend and husband, John.
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