Krabbe Disease
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CLINICAL_TRIALS trial_result · 2026-03-06Clinical Trial Update: Diffusion Tensor Imaging (DTI) in Infants With Krabbe Disease
University of Pittsburgh is conducting a diffusion tensor imaging study in infants with Krabbe disease, and the trial is currently ACTIVE_NO…
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CLINICAL_TRIALS research · 2026-02-09Clinical Trial Update: Longitudinal Study of Neurodegenerative Disorders
This clinical trial update describes a longitudinal study of several neurodegenerative and lysosomal storage disorders. The study is current…
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CLINICAL_TRIALS research · 2025-11-20Clinical Trial Update: Gene Transfer Clinical Trial for Infantile and Late Infantile Krabbe Disease Treated Previously With HSCT [Phase 1, Phase 2]
A phase 1/2 gene transfer trial for Krabbe disease in infants previously treated with hematopoietic stem cell transplantation is currently a…
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CLINICAL_TRIALS trial_result · 2025-11-13Clinical Trial Update: Study of Safety, Tolerability and Efficacy of PBKR03 in Pediatric Subjects With Early Infantile Krabbe Disease [Phase 1, Phase 2]
The clinical trial update indicates the Phase 1/2 study of PBKR03 in pediatric subjects with Krabbe disease is currently suspended.
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CLINICAL_TRIALS research · 2025-10-23Clinical Trial Update: The Myelin Disorders Biorepository Project
This clinical trials update describes the Myelin Disorders Biorepository Project (NCT03047369) from Children’s Hospital of Philadelphia, cur…
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CLINICAL_TRIALS research · 2025-09-08Clinical Trial Update: UCB Transplant of Inherited Metabolic Diseases With Administration of Intrathecal UCB Derived Oligodendrocyte-Like Cells [Phase 1]
Phase 1 trial evaluating intrathecal umbilical cord blood-derived oligodendrocyte-like cells for several inherited metabolic brain diseases;…
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CLINICAL_TRIALS trial_result · 2025-09-05Clinical Trial Update: Long-term Follow-up Study to Evaluate Safety and Efficacy of FBX-101 in Krabbe Patients
The long-term follow-up study to evaluate safety and efficacy of FBX-101 in Krabbe disease has been terminated.
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CLINICAL_TRIALS research · 2025-02-10Clinical Trial Update: Krabbe Disease Global Patient Registry
ClinicalTrials.gov entry for a global Krabbe disease patient registry indicates ongoing recruitment.
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CLINICAL_TRIALS trial_result · 2025-01-30Clinical Trial Update: Gene Transfer Clinical Trial for Krabbe Disease [Phase 1, Phase 2]
Update on a gene transfer trial for Krabbe disease sponsored by Forge Biologics; the Phase 1/2 study is currently not recruiting.
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CLINICAL_TRIALS trial_result · 2018-03-08Clinical Trial Update: The Natural History of Infantile Globoid Cell Leukodystrophy
The document reports the completed status of a natural history clinical study on infantile Krabbe disease sponsored by Zymenex A/S.