Rosenau Family Research Foundation

03/20/2026

We would like to express our gratitude to GEMMA Biotherapeutics for being a Silver Sponsor of KTRN 2026! Support from our network is what makes these meetings possible. Thank you for playing an integral role in bringing this group of experts and leaders together to push the needle forward!

03/19/2026

We would like to express our gratitude to M6P Therapeutics for being a Bronze Sponsor of KTRN 2026! Support from our network is what makes these meetings possible. Thank you to M6P Therapeutics for playing an integral role in bringing this group of experts and leaders together to push the needle forward!

03/16/2026

KTRN 2026 is quickly approaching! We have an exciting lineup of speakers and workshops on the docket, and are anticipating some in-depth presentations and discussions that will help our community create a roadmap for Krabbe disease-related science and innovation through 2030 and beyond.

We would like to take the opportunity to express our gratitude to KrabbeConnect for being a Non-Profit Sponsor of KTRN 2026. Support from our network is what makes these meetings possible. Thank you to KrabbeConnect for playing an integral role in bringing this group of experts and leaders together to push the needle forward!

03/01/2026

It’s ! Did you know rare diseases are more common than you’d think? With more than 10,000 rare conditions, everyone likely knows someone impacted by a rare disease, including Krabbe disease and Cystic Fibrosis. Rosenau Family Research Foundation is proud to play a role in funding research that bridges the gap between what we currently understand and what is yet to be learned, striving to improve the lives of everyone impacted by these diseases. Learn about the vast and diverse rare community at and rarediseaseday.us.

02/24/2026

Rosenau Family Research Foundation is counting down to on February 28th! There is fantastic work being done to research treatments for rare diseases, with much progress yet to come. We are grateful to call the National Organization for Rare Disorders () a partner in our work to improve the lives of patients impacted by Krabbe disease and Cystic Fibrosis through research funding and disease advocacy. Learn more at rarediseaseday.us.

02/18/2026

Submit your sponsorship for KTRN 2026 by February 20th!

KTRN 2026 is taking place March 18-20 at the Hilton Minneapolis/Bloomington.

Learn more about the agenda and access the sponsor form at rosenaufoundation.org/wp-content/uploads/2026/02/KTRN-2026-Sponsorship-Brochure-FINAL.pdf

Please email [email protected] with any questions.

See you in March!

02/17/2026

RFRF grant recipient Chris Lee, PhD, was at WORLDSymposium 2026 earlier this month presenting his RFRF-funded work on a pre-clinical human cell model of Krabbe disease! This research fills a need for tools to understand pathogenicity of patients’ mutations and testing of potential therapeutics. You can learn more about his grant work with the Biomedical Research Institute of New Jersey at https://rosenaufoundation.org/lee-2025/.

02/04/2026

Rosenau Family Research Foundation (RFRF) would like to extend its congratulations to its 2025 class of grant recipients:

Changfan Lin - Caltech
Hara Levy, MD, MMSc - University of Wisconsin, Madison
Chris Lee, PhD - Biomedical Research Institute of New Jersey
Natalia Gomez-Ospina, MD, PhD - Stanford University
Venkata Sai Chaluvadi - University of Pennsylvania
Tom Brett, PhD - Washington University in Saint Louis
Robert Thompson Stone, MD - University of Rochester

The mission and vision of RFRF is improving the lives of patients impacted by Krabbe disease and Cystic Fibrosis through research funding and disease advocacy, and helping individuals live life undefined by Krabbe disease and Cystic Fibrosis.

To support this mission and vision, the foundation considers grant funding for basic science research, translational research, and clinical research that can lead to or enhance treatments and cures for Krabbe disease and Cystic Fibrosis. Programs that promote, through education and awareness, the expansion of Newborn Screening, Krabbe disease, and Cystic Fibrosis are similarly considered.

“The 2025 grant awards reflect our unwavering commitment to advancing research in Krabbe disease and Cystic Fibrosis,” said RFRF Executive Director Gabriel M. Cohn, MD, MBA. “Our 2025 grant recipients showcase the broad spectrum of work being done to push the needle forward towards finding effective treatments and improving the lives of patients living with these diseases.”

2025 grant recipients were notified of their award in late 2025, with projects spanning two to three years from the start of funding.

Each full grant application is evaluated by RFRF’s Scientific Advisory Committee (SAC) for alignment with RFRF’s goals and objectives, as well as the proposal’s scientific merit, significance, robustness, and feasibility (scientific, technical, and financial).

Only grant applications that address specific scientific question(s) or applications that address specific educational/awareness goal(s) and that are aligned with RFRF’s mission and vision are funded.

To learn more about this and past year’s grant recipients, visit rosenaufoundation.org/funded-grants.

01/30/2026

An interview with RFRF grant recipient Allison Bradbury about her grant work studying Krabbe disease dog models, her experiences getting research funding, and more.

Watch now: https://youtu.be/K1wAx-e-bME

Allison M. Bradbury, MS, PhD is a tenure track assistant professor in the Department of Pediatrics at The Ohio State University and Principal Investigator in the Center for Gene Therapy at Nationwide Children’s Hospital. She leads a R01-funded translational laboratory with the goal of improving the understanding of disease mechanisms resulting in rare pediatric neurologic disorders to develop safe and efficacious targeted gene therapy approaches.

Dr. Bradbury received her PhD in Biomedical Sciences from Auburn University (2009-2014) where her doctoral research was focused on the development of AAV gene therapy in a feline model of GM2 gangliosidosis. This therapy is now in human clinical trials. Successively, her postdoctoral research fellowship was completed at the University of Pennsylvania and supported by a NRSA F32 Fellowship and NIH K99/R00 Pathway to Independence grant. During her postdoctoral fellowship she conducted a comprehensive natural history study in the canine model of globoid cell leukodystrophy (Krabbe disease) and demonstrated therapeutic efficacy in this model for the first time by utilizing AAV gene therapy to target both central and peripheral nervous system disease, with successful translation to the clinic. In her independent laboratory, Dr. Bradbury continues to optimize and translate next generation gene therapies for Krabbe disease.

Learn more at https://rosenaufoundation.org/bradbury-2024/.

An interview with RFRF grant recipient Allison Bradbury about her grant work studying Krabbe disease dog models, her experiences getting research funding, an...

01/16/2026

🎉 EEEK — WE’RE SO EXCITED!!! 🎉

After 2 years in the making, the full article is now published: Caregiver-reported disease burden in Krabbe disease: evaluating outcomes of hematopoietic stem cell transplantation 🧬

🔗 Read the full article: https://link.springer.com/article/10.1186/s13023-025-04176-3

A HUGE shout-out to the families who shared their experiences to help shape a better future for those impacted by .

We are deeply grateful to Rosenau Family Research Foundation for funding this work, Engage Health Inc. for conducting caregiver interviews and collecting the data, and United Leukodystrophy Foundation and Partners for Krabbe Research for helping raise awareness and amplify this important study. 💜

This publication is packed with important data families, clinicians, and states can use to better understand real-world outcomes of HSCT and inform newborn screening and treatment decisions.

✨ 3 Key Highlights:
• Early HSCT is associated with reduced disease burden and better overall quality-of-life scores compared to late or no transplant.
• Caregivers of early-transplanted infants reported improvements in sleep, mental health, and family/social relationships.
• The study provides critical evidence for states evaluating Krabbe disease newborn screening programs and understanding HSCT’s impact on families.

📌 Most importantly, this work will help families newly navigating a Krabbe diagnosis make more informed decisions about hematopoietic stem cell transplantation with data grounded in caregiver experience and patient-centered outcomes.

Let’s share this far and wide — real insights that matter to our community! 💜✨

01/16/2026

Amazing news! We are so honored to have funded this work.

🎉 EEEK — WE’RE SO EXCITED!!! 🎉

After 2 years in the making, the full article is now published: Caregiver-reported disease burden in Krabbe disease: evaluating outcomes of hematopoietic stem cell transplantation 🧬

🔗 Read the full article: https://link.springer.com/article/10.1186/s13023-025-04176-3

A HUGE shout-out to the families who shared their experiences to help shape a better future for those impacted by .

We are deeply grateful to Rosenau Family Research Foundation for funding this work, Engage Health Inc. for conducting caregiver interviews and collecting the data, and United Leukodystrophy Foundation and Partners for Krabbe Research for helping raise awareness and amplify this important study. 💜

This publication is packed with important data families, clinicians, and states can use to better understand real-world outcomes of HSCT and inform newborn screening and treatment decisions.

✨ 3 Key Highlights:
• Early HSCT is associated with reduced disease burden and better overall quality-of-life scores compared to late or no transplant.
• Caregivers of early-transplanted infants reported improvements in sleep, mental health, and family/social relationships.
• The study provides critical evidence for states evaluating Krabbe disease newborn screening programs and understanding HSCT’s impact on families.

📌 Most importantly, this work will help families newly navigating a Krabbe diagnosis make more informed decisions about hematopoietic stem cell transplantation with data grounded in caregiver experience and patient-centered outcomes.

Let’s share this far and wide — real insights that matter to our community! 💜✨