Year: 2025

Study: Progression of Spinal Cord Disease in Adult Men With ALD

Research / January 22, 2025

The article, Progression of Spinal Cord Disease in Adult Men with Adrenoleukodystrophy, details the results of a long-term study on the progression of spinal cord disease in adult men with ALD. The study, which followed 79 patients over seven years, used clinical tools like the EDSS, SSPROM, and 6MWT to measure disease progression. It showed …

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Lentivirus-modified hematopoietic stem cell gene therapy for advanced symptomatic juvenile MLD: a long-term follow-up pilot study

Research / January 22, 2025

The study “Lentivirus-modified hematopoietic stem cell gene therapy for advanced symptomatic juvenile metachromatic leukodystrophy: a long-term follow-up pilot study” investigates a pioneering treatment for juvenile metachromatic leukodystrophy (MLD), a rare, inherited neurological disorder caused by a deficiency in the ARSA enzyme. This enzyme deficiency leads to the toxic accumulation of sulfatides, causing progressive demyelination (damage …

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