ULF received donations in memorial or honorarium for the following during the months of October through December 2024. We apologize for any inadvertent omittance or misspellings. Please notify us if we have made a mistake at [email protected] or by calling 815-748-3211 In honor of Adam Sledzik Stanislaw Sledzik CTX is very bad ,rare disease. Adrianna …
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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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 …
