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Help Us Continue Building Bridges and Breaking Barriers for RUNX1-FPD
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At RRP, we are dedicated to improving the quality of life and preventing blood cancer for RUNX1-FPD patients. This mission relies on the strength of our community. Your support allows us to connect patients and researchers, fund groundbreaking research, and advance educational initiatives that bring hope to those with RUNX1-FPD.
Every gift matters. Please consider making a donation today—your generosity, no matter the size, drives meaningful change, and helps us continue building a brighter future for RUNX1-FPD patients and families. |
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Attendees of the 8th Annual RUNX1 Scientific Conference in Princeton, NJ. |
Thank You for Making the 8th Annual RUNX1 Scientific Conference a Success! |
We are immensely grateful to the 80+ attendees who joined us in Princeton for the 8th Annual RUNX1 Scientific Conference this year! It was a memorable gathering of brilliant minds and passionate advocates, all dedicated to advancing research and treatment options for RUNX1-FPD.
A highlight of the conference was the inspiring keynote address by John Crowley, the President & CEO of Biotechnology Innovation Organization (BIO). If you missed his talk or would like to revisit the impactful insights he shared, please scroll down to the "Patient & Family Updates" section of this newsletter for a link to the video. |
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We’re thrilled to announce that the 9th Annual RUNX1 Scientific Conference will be held virtually on Monday, September 8th and Tuesday, September 9th, 2025.
We’re equally excited to share that the virtual Patient Meeting will take place on Friday, September 19th, 2025.
We look forward to bringing the RUNX1-FPD community together for these inspiring and informative events. Stay tuned for more details about how you can participate next year! |
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- Dr. Laura Babbitt — RUNX1 Award of X-Cellence: Most Valuable Patient of the Year
This award celebrates the efforts of a dedicated individual who has excelled in advocacy leadership for RUNX1-FPD patients and families, as well as helping to further awareness about RRP.
- Janna Pelle — X-Out Cancer Challenge Campaign: Top Fundraiser Award
This award recognizes the exceptional achievement of being the top fundraiser in this annual campaign, demonstrating unwavering commitment and inspiring participation across our community.
- Dr. Ximena Jordan-Bruno — RUNX1 Award of X-Cellence: Clinician of the Year
This award is given each year to a clinician who has gone above and beyond to make an impact on RUNX1-FPD patient care, raise awareness about this disease and commit to collaboration with RRP.
- Dr. Lucia Castilla — RUNX1 Award of X-Cellence: Scientist of the Year
This award is received by a scientist who has made an outstanding contribution to our understanding of RUNX1-FPD and/or RUNX1 biology.
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Fundraising Update: Special Thanks to D.J., Taylore and Redwood Logistics
for Their Generous Gift! |
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In our October newsletter, we highlighted the inspiring efforts of Taylore and her fiancé, D.J., who organized a fundraiser at D.J.'s company, Redwood Logistics.
We are thrilled to announce that their initiative, coupled with a matching donation from Redwood Logistics, has culminated in a remarkable total of $32,500! We extend our heartfelt gratitude to Taylore, D.J., their fundraising team, Redwood Logistics, and all the donors who contributed to this incredible achievement. |
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Your generosity will help us continue to make a difference in the lives of individuals with RUNX1-FPD and their families! |
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RRP Joins National Effort to Advocate for Vital Legislation
Impacting Rare Disease Patients |
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RRP is proud to have joined 136 other organizations involved in American healthcare in signing a letter urging Congressional leaders to pass three critical bills:
These bipartisan bills are crucial for enhancing access to life-changing and lifesaving treatments for patients with rare diseases. Click on each bill's name above to learn more about the specific impacts and details of the legislation |
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Apply Now For The 2025 RRP-ALSF Early Career Investigator (ECI) Grant
Application Deadline: Thursday, December 19th by 8 p.m. EST
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This ongoing grant partnership between RRP and Alex’s Lemonade Stand Foundation supports early-career scientists focused on inherited hematologic malignancy predisposition disorders. Funding is awarded to projects designed to prevent the progression of pre-malignancy to hematologic malignancy in RUNX1-FPD patients.
The application deadline is December 19th, 2024 by 8 p.m. EST. Click the button below and scroll down the webpage for more details on applying:
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Discover What's New with the RUNX1 Patient Data Hub |
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We’re excited to announce significant upgrades to the RUNX1 Patient Data Hub (RUNX1 PDH), our secure online platform dedicated to serving the RUNX1-FPD community. The transition to the Matrix platform brings enhanced features designed to improve user experience: -
User-Friendly Interface: Simplifies data entry, making it easier to share information.
- Symptom Tracker: Allows you to monitor your health regularly.
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Electronic Health Records Integration: Offers streamlined storage of your health information in one location.
Consider Joining Today!
By sharing your health and family medical history, you play a vital role in enhancing our collective understanding and improving treatment approaches for RUNX1-FPD. Here’s how your participation makes a difference: -
Advance Research: Your experiences provide valuable insights that propel RUNX1-FPD research toward new treatments.
- Support Patient Care: Your anonymized data helps healthcare providers refine care strategies.
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Empower Advocacy: Your responses help us understand patient challenges, and tailor resources and educational efforts effectively.
If you decide to join*, meet the eligibility requirements and complete your enrollment, we'd like to honor your time with a $50 gift card. For more details or assistance, feel free to contact Amanda Eggen at aeggen@runx1-fpd.org or Lauren Bartlett at patientdatahub@runx1-fpd.org.
To learn more about the RUNX1 Patient Data Hub, click here.
To enroll in the RUNX1 Patient Data Hub, click here.
*PLEASE NOTE: If you previously enrolled in the RUNX1 Patient Data Hub, your data has been completely migrated to the Matrix system. Just click here, enter the email address you used to sign up originally and select “Forgot your password?” to set up your new password and access the platform.
If you have any questions, contact Dr. Amanda Eggen at aeggen@runx1-fpd.org. |
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Highlighting Our Scientific Conference Keynote Speaker: John Crowley |
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We were honored to have John Crowley as our keynote speaker at this year’s conference. John's remarkable journey in biotechnology began when his children were diagnosed with Pompe disease, leading to his heroic efforts to save their lives—an inspiring story that was adapted into a film starring Harrison Ford, Brendan Fraser and Keri Russell.
His keynote address, intertwined with personal anecdotes and professional |
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insights perfectly matched our conference theme, "Building Bridges, Breaking Barriers." To hear John's powerful message and learn more about his transformative work, click here to watch the recording. |
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On Friday, November 22nd, RRP’s Executive Director, Dr. Katrin Ericson, and RRP's Scientific Advisory Board Chair, Dr. Nancy Speck, summarized the key points of the RUNX1 research progress presented by scientists at this year’s 8th Annual RUNX1 Scientific Conference.
The presentation is intended for patients and family members who would like to understand the scientific progress achieved over the last year for RUNX1-FPD. Click the button below to watch the recording:
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New Insights on RUNX1 Controls the Development of Red Blood Cells and Platelets |
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Research by Dr. Diane Krause and her team at the Yale School of Medicine studied how the transcription factor RUNX1 plays a crucial role in determining the lineage specification of megakaryocytic-erythroid progenitors (MEPs). These progenitors can differentiate into either megakaryocytes or erythroid cells. Their study revealed that phosphorylation (addition of a molecule called phosphate) of RUNX1 by cyclin-dependent kinase 9 (CDK9) promotes the |
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differentiation of MEPs into megakaryocytes (cells that make platelets), while inhibition of this phosphorylation results in increased erythroid differentiation.
This discovery provides important insights into the mechanisms regulating megakaryocyte and red blood cell development and highlights the role of RUNX1 phosphorylation in controlling lineage fate. These findings may contribute to understanding and developing new approaches for treating conditions like RUNX1-FPD, where blood cell development is disrupted.
To read the full article in Blood click here: https://doi.org/10.1182/blood.2024023963 |
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Join us in the mission to prevent blood cancer and bring life-changing treatments to RUNX1-FPD patients. Every dollar you give goes directly to research and education—100%, no exceptions. Your support today could lead to tomorrow’s breakthroughs.
Make your gift now and be part of the progress. Together, we can make a difference! |
Thanks for following our progress and being a part of the RRP community!
Visit www.runx1-fpd.org to learn more and keep up-to-date between newsletters. |
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