KRABBE DISEASE
Clinical trials for KRABBE DISEASE explained in plain language.
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Gene therapy after stem cell transplant shows promise for rare brain disease
Disease control OngoingThis study tests a one-time gene therapy infusion (FBX-101) given after a standard stem cell transplant in 6 children with infantile Krabbe disease, a severe genetic disorder affecting the nervous system. The therapy uses a harmless virus to deliver a working copy of the GALC gen…
Matched conditions: KRABBE DISEASE
Phase: PHASE1, PHASE2 • Sponsor: Forge Biologics, Inc • Aim: Disease control
Last updated Jun 27, 2026 13:00 UTC
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Gene therapy boost for krabbe patients after transplant
Disease control OngoingThis early-phase trial tests a single infusion of a gene therapy called FBX-101 in 9 people with Krabbe disease who have already received a stem cell transplant. The therapy uses a harmless virus to deliver a working copy of the GALC gene, aiming to improve motor function and saf…
Matched conditions: KRABBE DISEASE
Phase: PHASE1, PHASE2 • Sponsor: Forge Biologics, Inc • Aim: Disease control
Last updated Jun 27, 2026 07:59 UTC
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Newborn screening study aims to catch rare diseases at birth
Diagnosis OngoingThis study offers voluntary screening for newborns in North Carolina to detect a wide range of rare health conditions early. Using a small blood sample already collected at birth, the program tests for dozens of disorders, including spinal muscular atrophy, cystic fibrosis, and m…
Matched conditions: KRABBE DISEASE
Sponsor: RTI International • Aim: Diagnosis
Last updated Jul 02, 2026 02:00 UTC
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Brain scan breakthrough could save babies from rare disease
Diagnosis OngoingThis study looks at whether a special brain scan called diffusion tensor imaging (DTI) can help doctors find Krabbe disease in newborns and decide who needs urgent treatment. About 100 babies with a positive newborn screen or family history of Krabbe disease will be scanned and f…
Matched conditions: KRABBE DISEASE
Sponsor: University of Pittsburgh • Aim: Diagnosis
Last updated Jun 27, 2026 09:11 UTC