MeSH tree: C18.452.648.189.362
A Northwestern Medicine study has uncovered a promising new therapeutic approach for a rare genetic brain disorder, according to findings published in Nature Communications.
Jan 15, 2026
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Some brain disorders are straightforward, such as the direct frontal lobe assault of a concussion or traumatic brain injury. Others, like Alexander disease, are akin to guerrilla warfare. Patients suffering from this genetic ...
Dec 1, 2025
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If administered early, gene therapy has the potential to change the medical history of children born with metachromatic leukodystrophy (MLD), a rare and lethal neurodegenerative disease of genetic origin which leads to the ...
Apr 24, 2025
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In a preclinical study, researchers at Children's Hospital of Philadelphia (CHOP) demonstrated a novel gene therapy with potentially increased effectiveness and safety for the treatment of metachromatic leukodystrophy (MLD), ...
Mar 12, 2025
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RCCS San Raffaele Scientific Institute in Milan researchers have discovered that hematopoietic stem cells (HSCs) adapt their lineage commitment during gene therapy based on the underlying genetic disease.
When the myelin sheath that surrounds nerve fibers in the brain and spinal cord becomes damaged, a number of debilitating conditions can result that limit mobility, inhibit independence and reduce life expectancy. Multiple ...
Jun 6, 2024
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Metachromatic leukodystrophy is a rare genetic disorder that mainly affects young children and results in severe neurological symptoms accompanied by a loss of motor and intellectual capacities. At Paris Brain Institute, ...
Apr 22, 2024
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In a joint effort with RIKEN BioResource Research Center and Mitsubishi Tanabe Pharma Corporation, a team of researchers led by Dr. Haruhisa Inoue (Professor, Department of Cell Growth and Differentiation, CiRA) improved ...
Apr 16, 2024
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The U.S. Food and Drug Administration has approved Orchard Therapeutics' Lenmeldy (atidarsagene autotemcel) as the first gene therapy for the treatment of children with metachromatic leukodystrophy (MLD).
Mar 25, 2024
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It can be a hassle to get to the doctor's office. And the task can be especially challenging for parents of children with motor disorders such as cerebral palsy, as a clinician must evaluate the child in person on a regular ...
Sep 14, 2023
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