MeSH tree: C18.452.584.563.641.803
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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Cancer cells seldom start off stealthy. Quite to the contrary, they announce their presence to the immune system by planting chemical red flags right on their membranes. Once alerted, the body's defenses can swoop in, destroying ...
Sep 9, 2024
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About 37 million people in the United States have chronic kidney disease and studies show that genetics may explain between 10% and 20% of cases in adults (and as many as 70% of cases in children).
Jul 29, 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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Chronic kidney disease (CKD) is extremely prevalent among adults, affecting over 800 million individuals worldwide. Many of these patients eventually require therapy to supplement or replace kidney functions, such as dialysis ...
Apr 3, 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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A gene variant found almost exclusively in the genomes of people of African ancestry increases the risk of developing Parkinson's disease, according to an international study of nearly 198,000 participants with this genetic ...
Aug 24, 2023
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When a patient experiences the first symptoms of Parkinson's disease, the disease has been developing for a long time, and the patient may have already lost half of a specific type of nerve cells in the brain.
Apr 25, 2023
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Scientists may have solved the question of why Ashkenazi Jews are significantly more susceptible to a rare genetic disorder known as Gaucher disease—and the answer may help settle the debate about whether they are less susceptible ...
Feb 6, 2023
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