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Article Abstract
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| http://www.ncbi.nlm.nih.gov/pmc/articles/PMC11534747 | PMC |
| http://dx.doi.org/10.21037/atm-24-63 | DOI Listing |
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Article Synopsis
- - Volanesorsen is an RNA therapy that has shown effectiveness in reducing triglyceride (TG) levels in a 13-year-old girl with lipoprotein lipase (LPL) deficiency, a rare disorder that can lead to severe complications like pancreatitis.
- - The study observed that after starting weekly injections of Volanesorsen, TG levels dropped significantly, with no hospitalizations for pancreatitis in the first 14 months, unlike the patient's previous 21 admissions over a similar timeframe.
- - The treatment was well-tolerated, highlighting an improved quality of life for both the patient and her family, and suggesting the need for broader access to Volanesorsen for pediatric patients with LPL deficiency.
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