Records containing: X-ALD
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CLINICAL_TRIALS study results posted · 2026-08-26A Phase 1/2 Randomized, Blinded, Dose-escalation Study to Evaluate the Safety and Efficacy of Intrathecal Administration of AAV9-ABCD1 Gene Therapy (SBT101) in Adult Patients With Adrenomyeloneuropathy
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CLINICAL_TRIALS study record update · 2025-10-31A Phase 1/2 Randomized, Blinded, Dose-escalation Study to Evaluate the Safety and Efficacy of Intrathecal Administration of AAV9-ABCD1 Gene Therapy (SBT101) in Adult Patients With Adrenomyeloneuropathy
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CLINICAL_TRIALS study record update · 2025-10-30Prospective, Retrospective, Multicenter, Observational Study of Disease Progression in Adults With Inherited Forms of Spastic Paraplegia
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CLINICAL_TRIALS study record update · 2025-10-23The Myelin Disorders Biorepository Project and Global Leukodystrophy Initiative Clinical Trials Network
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CLINICAL_TRIALS study record update · 2025-06-15LeukoSEQ: Whole Genome Sequencing as a First-Line Diagnostic Tool for Leukodystrophies
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CLINICAL_TRIALS study record update · 2021-08-11Multi-center Clinical Study on the Diagnosis and Treatment Management of Rare Neurological Disease in Children