Microvillus inclusion disease (MVID) is a rare type of congenital enteropathy in infants that causes devastating diarrhea and an inability to absorb food. Infants can lose liters of fluid a day, become severely dehydrated, ...
Mar 6, 2024
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Using a novel methodology, researchers at The Hospital for Sick Children (SickKids) are the first in pediatric research to use data from an international real-world cohort to overcome the barriers associated with conducting ...
Feb 29, 2024
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Researchers from the UCLA Health Jonsson Comprehensive Cancer Center have shown for the first time that a gene usually linked to giant axonal neuropathy, a rare and severe neurological condition, also plays a role in inhibiting ...
Feb 29, 2024
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An international team of scientists has developed a technology based on artificial intelligence (AI) for the study of minority diseases and has successfully applied it to identify the possible causes of the appearance of ...
Feb 28, 2024
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The success of mRNA vaccines against COVID-19 has unleashed a flood of interest in using the technology to create more vaccines and treatments for everything from rare diseases and infections to cancer.
Feb 28, 2024
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An international team has identified a new therapeutic for patients with a rare autoimmune disease called eosinophilic granulomatosis with polyangiitis (EGPA). A biologic drug called benralizumab has been shown to be non-inferior ...
Feb 23, 2024
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In an article published in the Journal of Pediatrics, researchers based in Brazil describe the case of a nine-year-old boy admitted to hospital with multiple symptoms and overlapping conditions that made diagnosis difficult, ...
Feb 19, 2024
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The U.S. Food and Drug Administration (FDA) has awarded Orphan Drug Designation to Canget BioTekpharma LLC for FL118, a drug candidate developed at Roswell Park Comprehensive Cancer Center, as a possible treatment for pancreatic ...
Feb 5, 2024
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The epigenetic 'signature' of a rare, hard-to-diagnose breast tumor has been found by scientists at the Garvan Institute of Medical Research. The discovery could lead to improved treatment guidelines and better outcomes for ...
Feb 2, 2024
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Treatment with a molecule known as A485 can quickly and temporarily increase levels of white blood cells, a critical part of the body's immune system, an effect that is difficult to deliver with currently available pharmaceuticals, ...
Jan 31, 2024
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