Welcome to KrabbeConnect
KrabbeConnect is dedicated to improving care and finding a cure for Krabbe disease through collaboration across a network of patients, advocates, doctors, researchers, and organizations. Our goal is to bridge science and patient knowledge to improve care and find a cure. We are committed to a patient-centered approach, ensuring that the voices of patients and caregivers guide research and care.
Our Heroes
We are working towards a day in which each patient receives an early diagnosis, has access to state-of-the-art care, and lives a life free of Krabbe disease. Help us celebrate all of the Krabbe disease Heroes and join the fight to #curekrabbe!
Current & Upcoming Events
September 2026
2026 Leukodystrophy & Newborn Screening Awareness Month
September is LeukodystrophyThe leukodystrophies comprise a group of progressive, genetic disorders mainly affecting the central nervous system (CNS). Most leukodystophies result from a disruption of the growth of the myelin she and Newborn Screening Awareness Month! The goal is to encourage parents, healthcare providers, and policymakers to advocate for the inclusion of more conditions, like leukodystrophy, in routine newborn screening panels. Read More
ONGOING
Be the Story — Krabbe Disease Newborn Screening Campaign
Are you interested in being the face of our Krabbe disease newborn screening state fact sheets? We’re on a mission to help bring more education and awareness to Krabbe disease newborn screening. All 50 states must be testing for this condition now to prevent a delayed diagnosis, especially in the most severe forms. You can be the story to help change Krabbe disease. See which states across the USA do not have a Krabbe disease fact sheet. If your state is not listed, we invite you to share your story. Read More
🎄❤️ Holiday Cheer for Krabbe Disease Families ❤️🎄
The holiday season can be a time of joy and celebration, but we also recognize that it can be especially difficult for families navigating the challenges of Krabbe disease. When families are carrying so much, the holidays can bring a mix of emotions, added stress, and reminders of just how much they have weathered.
We believe every family deserves to feel seen, supported, and remembered during this season. That’s why we’re excited to share that we will once again be offering our Holiday Cheer Program for families currently navigating Krabbe disease. ❤️
Our hope is to bring a little extra comfort, joy, and holiday magic to these incredible families as they navigate a journey no family should have to face alone.
✨ More information about the Holiday Cheer Program, including details on how to participate, will be available on our website soon! Stay tuned for updates.
Because even in the hardest seasons, a little kindness can make a meaningful difference. ❤️🎁
#KrabbeDisease #HolidayCheer #KrabbeWarriors #FamilySupport #hopeandlove
3 CommentsComment on Facebook
Last month, we hosted a webinar about KrabbeCURES to help our community better understand why participation matters—and how your information can make a difference.
Why join KrabbeCURES?
1. Your Experience Matters - Every Krabbe journey adds another piece to the puzzle and helps us build a clearer picture of the disease.
2. More Families = Better Data
The more people who participate, the stronger and more meaningful our data becomes. And when you participate, you can see real-time community data too—for example, how many people impacted by Krabbe are from Indiana or how others in the community answered certain questions.
3. Your Data Can Move Research Forward - Together, our information can help researchers identify patterns, understand gaps in care, ask better research questions, and work toward improved treatments—and ultimately, a cure.
One family’s experience is powerful. Hundreds of experiences together can change what we know about Krabbe disease.
👉 Join KrabbeCURES. Add your experience. Strengthen the data. Help move research forward here - youtu.be/eA-RaIgxVeA?si=-Nn5ZlyAENlX1H65.
#KrabbeCURES #krabbeconnect #curekrabbe #raredisease
0 CommentsComment on Facebook
🛍️ SHOP. EAT. RALLY. FOR A REASON. 💙
We’re bringing our community together for a day of shopping, food, connection and purpose — all while helping families impacted by Krabbe disease.
Come shop local craft vendors, grab something delicious from our food trucks, and learn more about why finding better treatments and ultimately a cure for Krabbe disease matters so much.
Admission is FREE for attendees!
And we’re looking for vendors to join us!
🛍️ Craft Vendors: $40
🚚 Food Truck Vendors: $120
Every vendor, every shopper, every meal purchased and every person who shows up helps us create awareness and rally around children and families facing this devastating disease.
Come for the shopping. Come for the food. Stay for the mission. Together, we can help give families hope for a future without Krabbe disease.
0 CommentsComment on Facebook
Help us welcome our newest board member - Lee Neumann - she holds a bachelor’s degree in Biology from Gustavus Adolphus College and a Juris Doctor from Hamline University School of Law. After several years practicing patent litigation at a large law firm in Minneapolis, Lee moved to a manufacturer and service provider for medical and surgical equipment. There, she is General Counsel and Corporate Secretary. Lee lives in Minnesota with her husband Eric and their two sons. We are so excited to have her part of the team!
2 CommentsComment on Facebook
Today marks the final day of Leukodystrophy & Newborn Screening Awareness Month. 🧡💙
Throughout September, we’ve shared information about Krabbe disease, the importance of newborn screening and early detection, and—most importantly—the voices and experiences of families who live with the realities of this disease every day.
We’ve talked about the challenges. We’ve celebrated progress. We’ve shared hope. And we’ve reminded people why early identification, research, resources, and connection matter.
To everyone who read a post, shared a story, participated in an event, donated, advocated, or simply took a moment to learn about Krabbe disease—thank you for joining us. Every share helps someone new understand this disease, and awareness creates connection.
September may be ending, but our work does not. Until there’s a cure, we won’t stop.
Thank you for standing with our Krabbe families—not just this month, but all year long. 🧡💙
Together, we continue the work to #CureKrabbe.
0 CommentsComment on Facebook
In honor of Leukodystrophy & Newborn Screening Awareness Month 🧡💙
This is what early identification can look like.
Meet twins Brendan and Trevor. They were diagnosed with Krabbe disease through Pennsylvania’s newborn screening program—before the disease had the opportunity to cause the devastating progression so many families have experienced.
Their early diagnosis gave their family something incredibly important: time and options. They were able to quickly connect with Krabbe specialists, undergo stem cell transplantation, and later participate in the investigational FBX-101 gene therapyA type of therapy that offers hope and promise for a cure for many genetic disorders. A working copy of the gene replaces the non-working copy of the gene. Gene therapy is at the forefront of many tr clinical trialA clinical trial is research designed to understand the safety and efficacy of a drug, biologic or device. There are 4 phases to most clinical trials from Phase 1 that seeks to answer safety concerns. The boys were among the first children treated in the trial’s low-dose cohort. Early clinical reports from that cohort described increased GALCA specific enzyme that receives its instructions from the GALC gene. When the GALC gene isn’t working properly, the enzyme, galactocerebrosidase, is unable to break down certain fats called galactolip More activity, reduced psychosineDue to the enzyme deficiency in Krabbe disease, psychosine accumulates in the nervous system of affected individuals. Psychosine, a galactolipid, is a cytotoxic type of lipid, that has destructive pro, and encouraging motor and brain-development findings.
Today, their parents describe them as doing very well physically—and their story is a powerful reminder of why we fight for newborn screening.
Early identification creates time.
Time creates options.
And options create hope.
This is why newborn screening matters. 🧡💙
#krabbedisease #newbornscreening #leukodystrophyawareness #krabbeconnect
4 CommentsComment on Facebook