MeSH tree: C18.452.648.595
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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The UK regulatory authorities have approved the first ever trial of a revolutionary gene therapy for young children diagnosed with Hunter syndrome, a devastating rare lysosomal storage disorder.
Oct 30, 2023
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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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University of Michigan researchers have discovered that the same cellular mechanism involved in a form of cystic fibrosis is also implicated in a form of a rare disease called cystinosis.
Aug 28, 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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Artificial intelligence is becoming increasingly important in drug discovery. Advances in the use of Big Data, learning algorithms and powerful computers have now enabled researchers at the University of Zurich (UZH) to better ...
Jul 14, 2023
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When people think about Alzheimer's disease, they usually associate it with seniors who have had a long and fulfilling life.
Jun 21, 2023
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Up to 40% of rare diseases show facial alterations that enable researchers to identify some pathologies, and they can even help establish an early diagnosis. Historically, the visual evaluation and use of some classic anthropometric ...
May 10, 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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