Inherited metabolic disorders
Late-infantile and juvenile Krabbe disease
Later-onset Krabbe disease is a GALC-related disorder in which myelin damage begins after the earliest infantile period. It can affect walking, vision, coordination and other neurologic functions, with variable progression.
Other names and abbreviations
LOKD · later-onset GLD · GALC deficiency · Krabbe disease · globoid cell leukodystrophy · Later-onset Krabbe disease · Late-onset globoid cell leukodystrophy
Where transplant fits
Allogeneic transplantation may be considered for selected patients before major functional decline. An appropriate related or unrelated donor may be used, but eligibility and benefit depend on disease stage and the pace of progression.
Treatment depends on the exact diagnosis, disease stage, prior treatment and the person’s health. These categories are not estimates of donor demand.
Treatment at a glance
- Who it affects
- Begins later in infancy or childhood; age categories do not perfectly predict the disease course.
- Other treatment options
- Neurologic monitoring, mobility and rehabilitation support, nutritional care and treatment of symptoms remain important.
- Cells used for transplantation
- When transplantation is appropriate, the graft contains blood-forming stem cells from a suitable donor. Bone marrow, peripheral blood or cord blood may be selected according to the condition and transplant protocol.
Why the details matter
Evidence from presymptomatic infantile Krabbe disease cannot be transferred unchanged to later-onset disease. Transplantation may not prevent all later peripheral-nerve or motor disability.
Questions to bring to your care team
What is the exact diagnosis or subtype? What is the goal of each treatment option? If transplant is being considered, why does it fit this situation, which cells would be used and what are the alternatives?
Sources and further reading
- Krabbe Disease
GeneReviews, University of Washington / NCBI Bookshelf · Accessed 2026-09-05 - Inborn Errors of Metabolism and Osteopetrosis
EBMT Handbook · 2024-04-11
Understanding can become action.
Some patients need a blood stem cell donor. Others receive different treatment. Wherever your interest began, you can help JBF reach more people who may be able to donate.
Explore the official registry serving where you live. It explains who can join, how registration works and what donation involves.
Find your official registryIf joining is not right for you, a gift to the Jada Bascom Foundation supports education, outreach and referrals to official registries.
Donate to JBFKeep learning
Why matching is hard: an interactive leukemia story
More in inherited metabolic disorders. Sharing a group does not mean sharing a treatment plan.
