MeSH tree: C16.320.565.398.641.803.925
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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In two new papers, researchers from The Jackson Laboratory (JAX) report the successful use of two approaches—gene therapy and bone marrow transplantation—to alleviate symptoms of multiple sulfatase deficiency (MSD), an ultra-rare ...
Jan 29, 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.
Researchers from Children's Hospital of Philadelphia (CHOP) have tested a preclinical model for an experimental gene therapy designed to treat multiple sulfatase deficiency (MSD), a disorder that affects the brain, lungs, ...
Sep 9, 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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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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