ICYMI: Successful Phase 3 Study of Familial Chylomicronemia Syndrome Drug May Win Drug Approval in US
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ICYMI: Successful Phase 3 Study of Familial Chylomicronemia Syndrome Drug May Win Drug Approval in US

According to a publication from BioPortfolio, American biotechnology companies Akcea Therapeutics and Ionis Pharmaceuticals recently published final study results from their phase 3 clinical study of Waylivra (generic name volanesorsen)…

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A Potential Treatment for Familial Chylomicronemia Syndrome Performs Well in Trials

According to a story from EurekAlert!, the results of a recent clinical trial should get the attention of patients in the US with the rare disorder familial chylomicronemia syndrome. The…

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Study has Commenced for a New Potential Familial Chylomicronemia Syndrome Treatment

Hypertriglyceridemia Hypertriglyceridemia is the term used to describe abnormally high numbers of triglycerides in the blood. People with Hypertriglyceridemia have an increased risk of heart disease, stroke, and heart attack.…

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New Treatment for Familial Chylomicronemia Syndrome is Close to Approval in the EU

Familial Chylomicronemia Syndrome Familial chylomicronemia syndrome (FCS) is a condition that is considered ultra-rare. It's caused when the enzyme called lipoprotein lipase (LPL) is impaired. It is can result in…

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Importance of Patient-Centered Research in Familial Chylomicronemia Syndrome
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Importance of Patient-Centered Research in Familial Chylomicronemia Syndrome

Familial Chylomicronemia Syndrome (FCS) Is a rare disease caused by malfunctioning lipoprotein lipase. This results in a buildup of triglycerides in the body's plasma. FCS can cause pancreatitis, memory loss, nerve…

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ICYMI: After Rejection, The Familial Chylomicronemia Foundation Urges The FDA to Consider Approving New Treatment
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ICYMI: After Rejection, The Familial Chylomicronemia Foundation Urges The FDA to Consider Approving New Treatment

According to a story from BioSpace, the U.S. Food and Drug Administration (FDA) recently rejected the approval of the drug Waylivra, which was in development for the treatment of familial…

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Results from First Study on Patient-To-Patient Connectivity in Those with FCS

  According to DAIC, Akcea Therapeutics Inc., has recently announced their publication of results from their study analyzing patient-to-patient connectivity towards the management of the rare disease familial chylomicronemia syndrome (FCS).…

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Treatment for Familial Chylomicronemia Syndrome Gets Recommendation From FDA Committee
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Treatment for Familial Chylomicronemia Syndrome Gets Recommendation From FDA Committee

According to a story from cafepharma.com, Akcea Therapeutics, Inc. recently announced that the FDA's Division of Metabolism and Endocrinology Products Advisory Committee voted to recommend approval of the therapy Waylivra…

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Gene Therapy Appears Effective for Familial Chylomicronemia Syndrome
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Gene Therapy Appears Effective for Familial Chylomicronemia Syndrome

According to a story from eurekalert.org, the first patient that received treatment with alipogene tiparovovec reported positive results after an eighteen month treatment period. Alipogene tiparovovec is a gene therapy…

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Communicating and Connecting With Others Can Improve Quality of Life in FCS Patients
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Communicating and Connecting With Others Can Improve Quality of Life in FCS Patients

According to a story from the Rare Disease Report, the results of a recent survey questionnaire indicate that communicating with other rare disease patients can cause a significant improvement in…

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How this Rare Disease Mom’s Letter Offers Encouragement
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How this Rare Disease Mom’s Letter Offers Encouragement

According to an encouraging letter she wrote in The FCS Foundation, the day that Melissa learned that her beautiful 3-week-old daughter had a rare disease- familial chylomicronemia syndrome (FCS), or lipoprotein lipase…

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Global Genes Highlights Firefighter Experience with Familial Chylomicronemia Syndrome
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Global Genes Highlights Firefighter Experience with Familial Chylomicronemia Syndrome

Severe diarrhea. Daily abdominal pain. Extremely high triglycerides. Chronic pancreatitis. Joint pain. Incredibly uncomfortable conditions for anyone. Now imagine you’ve experienced this pain your entire life without knowing what was…

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Something Had to be the Stepping Stone
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Something Had to be the Stepping Stone

Medicine is expensive. Most patients aren’t millionaires. It’s disheartening that treatments which could be so beneficial are out of reach for so many. It’s even more disheartening when their production…

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