FOXG1 Research Foundation's Nasha Fitter Speaks At The White House Rare Disease Forum

FOXG1 Research Foundation Co-founder and CEO Nasha Fitter was the first spotlight speaker at the White House Rare Disease Forum hosted by the White House Office of Science and Technology Policy's Health Outcomes Team February 28th ahead of Rare Disease Day 2024.

Nasha Fitter represented the FOXG1 syndrome community as well as the 30 million patients, 15 million children, in the United States living with a rare disease. Nasha spoke about radical funding changes required to support drug development uniquely led by patient organization groups like the FOXG1 Research Foundation.

Following Nasha’s clear and impactful presentation, following speakers from ARPA-H, the Chan Zuckerberg Initiative, and Global Genes cited Nasha’s speech and the work of the FOXG1 Research Foundation to show how efficiently patient organizations can drive drug development if given funding.

Thank you to the White House for giving this platform, listening, and considering how far government funds can go to saving lives and alleviating significant economic burden if distributed among the patient organizations that are swiftly driving drug development because it is their children’s lives at stake

FOXG1 Research Foundation CEO and co-founder Nasha Fitter speaks at the first White House Rare Disease Forum

Nasha’s speech has sparked a conversation throughout the rare disease space.

See Nash’s LinkedIn follow up of the forum …

FOXG1 Research Foundation co-founder Nasha Fitter is a spotlight speaker at the White House Rare Disease Forum 2024

On Wednesday, February 28, as part of the Biden-Harris Administration’s goal to improve health outcomes, the White House Office of Science and Technology Policy will host a forum marking Rare Disease Day and highlighting the Biden-Harris Administration’s commitment to supporting patients and families facing a rare disease and delivering progress against the nearly 10,000 known rare diseases that impact up to 30 million Americans.
 
Leaders from the Biden-Harris Administration and the private and nonprofit sectors will work together to:

• Elevate the experiences and expertise of people and families facing rare diseases;

• Share developments from the Biden-Harris Administration and rare disease community and learn from one another to accelerate progress; and

• Identify opportunities to improve rare disease diagnosis, advance research and access to innovation to deliver effective treatment, and boost support for patients and families.

WHEN: Wednesday, February 28, 5:30 PM – 7:00 PM ET
WHERE: The event will be livestreamed at https://youtube.com/live/uG43oLZzeBE
FEATURED SPEAKERS: 

• Dr. Arati Prabhakar, Assistant to the President for Science and Technology, Director of the White House Office of Science and Technology Policy

• Dr. Danielle Carnival, Deputy Assistant to the President for the Cancer Moonshot, Deputy Director for Health Outcomes, White House Office of Science and Technology Policy

• Dr. Monica Bertagnolli, Director, National Institutes of Health

• Dr. Renee Wegryzn, Director, Advanced Research Projects Agency for Health

• Nasha Fitter, Co-founder, FOXG1 Research Foundation

• Emily Kramer-Golinkoff, Co-founder, Emily’s Entourage

• Dr. David Fajgenbaum, Co-founder, Every Cure

• Shonta Chambers, Executive Vice President-Health Equity Initiatives and Community Engagement at Patient Advocate Foundation

• Dr. Joni Rutter, Director, National Center for Advancing Translational Sciences

• Tamar Thompson, Vice President, Head of Global Corporate Affairs, Alexion, AstraZeneca Rare Disease

• Dr. Edward Neilan, Chief Medical and Scientific Officer, National Organization for Rare Diseases

• Charlene Son Rigby, Chief Executive Officer, Global Genes

• Tania Simoncelli, Vice President, Science and Society, Chan Zuckerberg Initiative

• Julia Tierney, Deputy Center Director (Strategy, Policy & Legislation), Center for Biologics Evaluation & Research, Food and Drug Administration

• Paul Melmeyer, Vice President, Public Policy and Advocacy, at the Muscular Dystrophy Association

• Kim McClellan, President of the Recurrent Respiratory Papillomatosis Foundation

• Annie Kennedy, Chief of Policy, Advocacy, and Patient Engagement, EveryLife Foundation

CONTACT: For press inquiries related to this event please email [email protected].
 
BACKGROUND: The Orphan Drug Act defines a rare disease as a disease or condition that affects fewer than 200,000 people. In the United States, rare diseases affect more than 30 million people in total. Of nearly 10,000 known rare diseases, only 5% have a Food and Drug Administration-approved treatment option.
Recent scientific and technological advancements have provided hope for understanding and delivery of new treatments for rare diseases, but significant work remains to address this critical public health issue that effects about 1 in 10 Americans.

FOXG1 Parents Support Webinar: “Tell Us Abey” Communications System

The FOXG1 Parents Support Team introduces Josh Weitzman, co-creator of the "Tell Us Abey" communications system that he and his wife Michelle developed for their son Abraham “Abey,” who has FOXG1 syndrome. Witnessing Abey's ability to communicate with this tool has given the FOXG1 community a new outlook on our children's  cognitive abilities. Abey is a student at Columbia University and a skilled creative writer. In this webinar, Josh shares more about Abey, their FOXG1 journey, and explains how "Tell Us Abey" works.

Be sure to read Abey’s blog post titled: My FOXG` story, which he wrote to introduce himself to the FOXG1 community. READ HERE

This webinar follows the incredible feedback after our co-founder Nicole Johnson wrote about her family’s experience when Abey met Josie. READ HERE

Abey’s articles that Josh talks about can be found HERE -just type Abraham Weitzman and they will all come up.

FOXG1 Parents, make sure you’re in the FOXG1 Research Parents Connect Facebook Group to learn about all the upcoming FOXG1 Parens Support Zooms and webinars.

Let us know what topics you’d like us to cover! [email protected]

Also, sign up for our updates at the footer of this website.

And follow us on social! @foxg1research #FOXG1 ResearchFoundation

COMBINED Brain Interviews FOXG1 Research Foundation Co-Founder, Nicole Johnson

For FOXG1 Awareness Month, Terry Jo Bichell, the Director of COMBINED Brain spoke to Nicole Johnson about her FOXG1 journey. Nicole discusses her daughter Josie, her family, and how the FOXG1 Research Foundation was created. Learn the FRF's research strategy and more.

One Rare Disease Mom and Patient Organization Leader to Another…

Terry Jo Bichelle is the founder of COMBINED Brain, The Consortium for Outcome Measures and Biomarkers for Neurodevelopmental Disorders that is devoted to speeding the path to clinical treatments for people with severe rare genetic non-verbal neurodevelopmental disorders by pooling efforts, studies and data. Nicole Johnson is on the board of COMBINED Brian and is the co-founder and Executive Director of the FOXG1 Research Foundation.

Terry Jo speaks to Nicole about her journey as a FOXG1 mom, from the early days of searching for her daughter Josie’s diagnosis to starting the FOXG1 Research Foundation. As Terry Jo says the FOXG1 Research Foundation has “really power packed” the work in four years to drive the science towards therapeutics for all individuals with FOXG1 syndrome.

"Inside Research with Nasha" - Repurposed Drug Screening for FOXG1

We’re kicking off a new series called “Inside Research with Nasha.”

Our FOXG1 Research strategy is to leave no stone unturned. We’re looking at every possible angle in science to find disease-modifying therapies and ultimately a cure for FOXG1 syndrome and we want to keep everyone informed along the way. We’re also taking this opportunity to really explain what so many buzz words in science mean and how they fit into the research projects we are funding.

Today, we’re very excited to talk about our small molecule drug screening projects. Nasha explains some of those buzz words that covid made mainstream, like high-throughput screening, repurposing, small molecules, and more.

You can find more information about our Research Projects, our Path to a Cure and our FOXG1 Science Team on our website.

Please follow us on social @FOXG1Research so you don’t miss any of our Inside Research updates!