Cancer specialists have taken a novel path to advance their research—tapping into a Facebook group for rare ovarian cancer insights.
Apr 6, 2023
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A clinical trial at the National Institutes of Health found that a medication, denosumab, significantly reduced abnormal bone turnover in adults with fibrous dysplasia, a rare disease marked by weak and misshapen bones. Bone ...
Apr 5, 2023
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Results from a clinical trial conducted by researchers at the National Institutes of Health (NIH) show that people with low-grade lymphomatoid granulomatosis who are treated with interferon alfa-2b, a type of immunotherapy, ...
Apr 4, 2023
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Providing an overview of the submissions process and examples of U.S. Food and Drug Administration (FDA) applications for Orphan Drug Designation (ODD) and Rare Pediatric Disease Designation (RPDD), a new article can help ...
Mar 27, 2023
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Statins are the most commonly used medication in lowering blood cholesterol, prescribed to tens of millions in the Western world. Statins act through inhibition of the enzyme HMG CoA reductase. Nearly 20% of statin users ...
Mar 21, 2023
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Using a new computational approach they developed to analyze large genetic datasets from rare disease cohorts, researchers at the Icahn School of Medicine at Mount Sinai and colleagues have discovered previously unknown genetic ...
Mar 16, 2023
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In times of crisis, it is advantageous for people with rare diseases to receive care in highly specialized centers. This is underlined by an MHH study. It shows that the people with the autoimmune diseases NMOSD and MOGAD ...
Mar 14, 2023
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US pharmaceutical giant Pfizer announced Monday that it had reached a deal to buy biotech firm Seagen, specializing in innovative cancer treatment, for $43 billion.
Mar 13, 2023
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When Dana Avellino, now 36, first noticed a lump near her groin in the summer of 2018, she thought it was related her recent cesarean section. Her younger daughter was only 2 months old at the time. When a biopsy revealed ...
Mar 9, 2023
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The movement disorder ADCY5-related dyskinesia can be treated with the asthma drug theophylline. This has been shown in a recent study by Martin Luther University Halle-Wittenberg (MLU), University Medicine Halle and University ...
Mar 6, 2023
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