2026 Newsletters

Insight Drop on the Diagnostic Journey for RUNX1-FPD 

Our latest analysis of the RUNX1 Patient Data Hub provides new insights into the diagnostic journey: how long it took, how many providers were involved, and what patients see as barriers that slowed the process down.

The findings reflect an experience many in our community know firsthand: getting answers with a rare disease takes persistence, and often takes far longer than it should.

If you or someone in your family is still seeking a diagnosis, or if you're curious how your experience compares to others in the community, this one is worth a read.

Wondering what you can do to help others find answers sooner? You can share RRP’s medical education webinar recordings with all of your clinical care teams and/or support RRP to do more of this educational programming to help the next generation of health care professionals to recognize the signs of RUNX1-FPD sooner.

Federal Funding Rule Could Affect Rare Disease Research: Act Before July 13

The Office of Management and Budget has proposed a rule that could allow political appointees to override peer review in federal grant funding decisions, permit active grants to be canceled mid-project, and restrict how researchers publish their findings.

RRP seeds early-stage RUNX1-FPD research, but federal funding is what takes that work to scale, and this proposal puts that pipeline at risk. 

You can read the full proposed rule at regulations.gov, Docket No. OMB-2026-0034 and submit a comment directly there before the July 13 deadline.

Watch for an email from RRP in the coming days with a template letter and step-by-step submission guide. Questions in the meantime? Reach out at info@runx1-fpd.org.

Note: this rule does not directly affect the NIH RUNX1 Clinical Research Study, but it does threaten the extramural grants that build on it.

Stem Cell Harvesting & Banking Clinical Trial

Many in our community ask whether there is anything they can do today to take a more proactive role in their future.

While there are not yet proven ways to prevent blood cancer in RUNX1-FPD, there may be steps individuals can take now to prepare for potential future treatment advances. One such option is banking your own stem cells while you are healthy, preserving them for the possibility of future cell-based therapies.

The Stem Cell Harvesting and Banking Clinical Trial, an RRP-sponsored study currently enrolling adults at both MD Anderson Cancer Center and Children's Hospital of Philadelphia, is exploring whether stem cells can be safely collected and stored from individuals with RUNX1-FPD for potential future use by those individuals.

One of the greatest hopes for these banked cells is that advances in gene editing may one day allow a person's own stem cells to be corrected and returned to them, potentially avoiding the need for a donor transplant.

You can learn more by reviewing the webinar featuring the doctor (Tim Olson, MD, PhD) leading the study at CHOP here.

For full details, a one-page study summary, and an interest form, visit our website through the link below. U.S.-based patients with questions about eligibility or travel support can contact Dr. Amanda Eggen.

International patients: we know participation is complicated right now and are actively working on solutions. Please reach out if you'd like to learn more.

Upcoming RRP Research Webinar

Wednesday, July 29th, 2026
12:00 p.m. PDT • 3:00 p.m. EDT •

8:00 p.m. BST

We have an exciting research webinar on the horizon with Dr. Weixin Tang, Associate Professor of Chemical Biology and Biophysics at the University of Chicago.

She will be joining us to speak about gene correction for germline RUNX1 mutations. This is an area with significant implications for the future of RUNX1-FPD treatment.

You can register for this webinar below:

AI Research Funding Opportunity

Anthropic announced the upcoming launch of Claude Science, an AI workbench that connects to more than 60 scientific databases, including PubMed, ClinVar, and GEO, and generates reproducible, auditable outputs.

For researchers working on RUNX1-FPD, the potential applications are real: single-cell and sequencing analysis, CRISPR screen design, variant interpretation, and more.

Anthropic is currently funding up to 50 research projects with up to $30,000 in credits each.

Applications are open through July 15, 2026. If this could accelerate your work, we encourage you to apply below:

RRP Research Webinar Roundup:

“Mechanisms underlying aging-associated increased heterogeneity and selection for clonal hematopoiesis mutations in hematopoietic stem cell pools.”

Thank you to Dr. James DeGregori for his insightful presentation and for sharing his time and expertise with attendees, and thank you to everyone who registered and attended our first RRP Research Webinar of 2026.

You can find a link to the final recording below:

RRP Medical Education Webinar Roundup:

“Recognizing Pediatric Hereditary Hematologic Malignancies”

Earlier this month, we hosted a MedEd webinar on recognizing hereditary hematologic conditions and practical strategies for pediatric oncology.

Thank you to Dr. Serine Avagyan for her presentation, and to Dr. Vivian Chang and genetic counselor Kayla Hamilton for joining the discussion.

If you missed it, the recording is now available: