KrabbeConnect

02/20/2026

💙🧡 **Rare Disease Day – February 28** 🧡💙

On February 28th, we join millions around the world to recognize **Rare Disease Day** — a day dedicated to raising awareness, advancing research, and standing alongside families navigating rare conditions like **Krabbe disease**.

At KrabbeConnect, we “Show Our Stripes” for:
🧬 Earlier diagnosis through newborn screening
💛 Stronger patient support and reduced medical burden
🔬 Innovative research and treatment development
🌎 A future where no family faces Krabbe alone

Krabbe disease may be rare, but our community is powerful. Together, we amplify voices, educate policymakers, support families, and push for progress.

📸 Download the “Show Your Stripes” template and tell us who you’re showing your stripes for.
Tag us and use

Because rare is many. And together, we are louder.

Link to download photo - https://bit.ly/4rudK6M

02/18/2026

For children identified through newborn screening as low-risk for late-onset Krabbe disease, progression in infancy is less likely — and follow-up can be thoughtfully tailored. We invite you to review the graphic below or check out the full publication here - https://pubmed.ncbi.nlm.nih.gov/33832819/

02/17/2026

Caregiving for a loved one with Krabbe disease impacts far more than medical appointments.

This figure from our recent publication highlights the emotional and social burdens caregivers experience — from disrupted sleep and mental health strain to challenges in relationships with spouses, friends, extended family, and even other children in the home.

Across groups, caregivers reported meaningful impacts on:
💤 Sleep
🧠 Mental health
❤️ Relationships
💪 Physical health

These numbers represent real families navigating isolation, stress, and constant vigilance — often while managing complex medical care decisions like transplant timing.

At KrabbeConnect, we believe caregiver voice matters. Data like this helps clinicians, researchers, and policymakers better understand the true burden of disease — and why comprehensive support systems are essential.

📖 Read the full peer-reviewed article here:
https://pubmed.ncbi.nlm.nih.gov/41501923/

Because behind every data point is a family doing everything they can. 💛

02/16/2026

Happy Monday!

Part 2: (TOMORROW) at 4:00 PM - 5:00 PM CST | 5:00 - 6:00PM EST

This panel-style conversation centers on real stories from individuals who have navigated IVF with PGD/PGT-M, creating space for shared learning, meaningful questions, and community connection.

👉 Register here for Part 2- https://us02web.zoom.us/.../register/EJmOeqz9QIiQptyvQyvQxA

02/11/2026

Our community could use a little fun—and a lot of hope. Join us for Putt-Putt to , where every putt helps support families impacted by Krabbe disease.

Learn, engage, and make a difference.
Register today here - https://krabbeconnect.org/kcevent/putt-putt-to-curekrabbe/

02/09/2026

While Grayson was in the hospital fighting for his health, his grandpa stepped up in another powerful way—by advocating for Medicaid so families like theirs can continue caring for medically fragile children at home.

Proposed cuts to Medicaid don’t just impact budgets—they impact real children, real families, and real lives. These programs allow kids like Grayson to receive the care they need at home, surrounded by family, rather than forcing already fragile systems into crisis.

When Medicaid is threatened, families lose critical home-based support, hospitals and care facilities become overwhelmed, and children face unnecessary disruption in their care. Advocacy like this matters—because no family should have to choose between being at a child’s bedside and fighting for the services that keep them safe.

Thank you to Grayson’s grandpa, and to all caregivers and advocates who raise their voices when it matters most. 💙

Protect Medicaid. Protect families. Protect kids like Grayson.

02/05/2026

Registration is OPEN! 🎉

Join us for a two-part webinar series on IVF with PGD/PGT-M—designed to help families understand their options and hear real-life experiences.

Part 1: (Feb 10th)- provides an overview of IVF with PGD/PGT-M, explaining how the process works, what genetic testing involves, and the important medical and emotional considerations families may face when navigating genetic risk.

👉 Register here for Part 1-https://us02web.zoom.us/meeting/register/79QCftJQRRidTTgEFq4oXQ

Part 2: (Feb 17th) - This panel-style conversation centers on real stories from individuals who have navigated IVF with PGD/PGT-M, creating space for shared learning, meaningful questions, and community connection.

👉 Register here for Part 2- https://us02web.zoom.us/meeting/register/EJmOeqz9QIiQptyvQyvQxA

02/05/2026

Exciting progress in the Krabbe disease community! Today we’re proud to share that Polaryx Therapeutics will be presenting late-breaking data on their lead investigational therapy, PLX-200, at the 22nd Annual WORLDSymposium as they prepare to launch the Phase 2 SOTERIA trial — including promising work in Krabbe disease.

At WORLDSymposium this week, KrabbeConnect had the opportunity to meet with the Polaryx team, and we’re energized by their commitment to advancing research that could one day impact families living with Krabbe disease. As the SOTERIA trial moves forward, we look forward to assisting Polaryx in navigating the unique challenges of Krabbe and helping ensure the voices of patients and families are part of the journey.

Polaryx to Present Late-Breaker Data Related to SOTERIA at the 22nd Annual WORLDSymposium™ as Company Prepares for SOTERIA Trial Launch February 3, 2026 Oral presentation provides additional scientific rationale on Krabbe disease, one of four LSDs in the Phase 2 SOTERIA basket trial designed to ev...

02/04/2026

💙 A historic step forward for babies with Krabbe disease 💙

North Dakota officially began newborn screening for Krabbe disease yesterday.

That brings us to 19 states ensuring babies have a chance at early detection—and a chance at life.

Early diagnosis saves lives. It gives families time, options, and hope where there was once only uncertainty.

We will not stop until every baby, everywhere has the same opportunity—no matter their zip code or country of birth.

Thank you to the advocates, clinicians, families, and supporters who continue to walk this journey with us. Together, we move closer to a cure.

02/04/2026

Amazing news! This is a big win for the rare disease community 💙.

‼️Breaking News: Rare Pediatric Disease PRV Program Reauthorized by Congress!

After a two-year campaign to reauthorize the Rare Pediatric Disease Priority Review Voucher (PRV) Program, the rare disease community’s relentless advocacy has paid off.

Congress has passed the Labor, HHS, and Related Agencies Appropriations bill, effectively reauthorizing the PRV Program for five years while also funding a number of other critical healthcare agencies.

We applaud the reauthorization of the PRV Program and renewed investments in critical health research and public health programs.

Thank you to the congressional champions who have partnered with our rare disease community to secure these advances. While significant work remains to enable all those living with rare diseases to thrive, today’s progress will accelerate innovation, expand access to life-changing therapies, and offer renewed hope to children and families whose futures once seemed beyond reach.

To learn more about the full healthcare package, please visit our website: https://everylifefoundation.org/congress-passes-five-year-reauthorization-of-rare-pediatric-disease-prv-program/

02/03/2026

Take a break from the cold and putt for a purpose!

Join us for the Puttshack Miniature Golf Tournament benefiting families impacted by Krabbe disease. Enjoy a fun night out with friends, great food, friendly competition, and putt-tastic prizes for our top mini-golf masters!

💙 Plus, meet the Wolford family, who will share their story and why this mission matters.

📅 March 20, 2026
🕠 Putt Start Time: 5:30PM
📍 Puttshack | Edina, MN
Register here: https://krabbeconnect.org/kcevent/putt-putt-to-curekrabbe/

Grab a team or sign up solo—every putt helps move research and support forward.

02/02/2026

If you’re attending WORLDSymposium 2026, be sure to connect with Stacy or Michelle to learn more about KrabbeConnect’s mission and work to improve outcomes for individuals and families impacted by Krabbe disease. Collaboration, conversation, and shared urgency move this field forward. Email us at info@krabbeconnect.org to schedule a meeting.