This week Ionis Pharmaceuticals, Inc. issued a press release via PRNewswire announcing topline Phase 3 results for its Balance study of olezarsen. The study ( NCT04568434 ) enrolled people with…
Right now, there are insufficient treatment options for familial chylomicronemia syndrome (FCS). Unfortunately, this means that individuals living with this rare genetic disease are left without much assistance. The…
PTC Therapeutics has just announced that their therapy Waylivra (volanesorsen), a treatment for familial chylomicronemia syndrome (FCS), has been given Category 1 classification from the Drug Market Regulation Chamber in…
According to an article in the Times Union, a group of parents with children who have been diagnosed with rare diseases are leading the crusade to foster federal legislation which…
Welcome to the Rare Classroom, a new series from Patient Worthy. Rare Classroom is designed for the curious reader who wants to get informed on some of the rarest, most…
On October 15th, 2020, the Rare Disease Legislative Advocates (RDLA) held a webinar discussing some of the latest news regarding ongoing legislation that is most relevant to the rare disease…
Happy Friday! July is almost over, which means that Juvenile Idiopathic Arthritis Awareness Month is drawing to a close. But it's not too late to bring attention to this often…
According to a story from msn.com, two women who were impacted by the rare disease familial chylomicronemia syndrome (FCS) have lead the charge in changing the procedures used by the…
In a July 9 press release, the Familial Chylomicronemia Syndrome (FCS) Foundation announced its support for a bill introduced by Congressman Paul Tonko (D-NY) and David B. McKinley (R-WV).…
According to a story from Rare Disease Report, there are several different rare diseases and conditions in which patients can find relief from most or all of their symptoms by…
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)…
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…
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.…
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…
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…
Heidi Jo Price, beautiful and brave, has jumped out of airplanes with a pack on her back as a member of the 173 Airborne Brigade Combat Team. What brought this…
Lindsey Sutton showed signs of familial chylomicronemia syndrome as early as five weeks old. Sutton is now 28 years old. Because of her condition, she must adhere to a very…
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…
According to a story from Akcea Therapeutics, the company recently received a reply from the US Food and Drug Administration (FDA) in regards to their New Drug Application for WAYLIVRA…
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).…
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…
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…
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…
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…
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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