Phase 3B Repatha Trial Shows Promise for Pediatric Patients with HeFH
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Phase 3B Repatha Trial Shows Promise for Pediatric Patients with HeFH

Between August 29 and September 1, researchers joined in at ESC 2020 - The Digital Experience, developed and organized by the European Society of Cardiology (ESC). During this online conference,…

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Heterozygous Familial Hypercholesterolemia Trial Shows Positive Results of Therapy in Teenagers

Researchers in Canada have just released positive results from their heterozygous familial hypercholesterolemia (HeFH) clinical trial. This investigation was examining the effect of Repatha on teenagers with the condition. The trial…

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ICYMI: Good Results From Evinacumab for Rare Form of Familial Hypercholesterolemia
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ICYMI: Good Results From Evinacumab for Rare Form of Familial Hypercholesterolemia

  A press release issued recently by Regeneron Pharmaceuticals heralded the acceptance of a Biologics License Application by the FDA for Priority Review of its investigational drug, evinacumab. The Priority…

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BLA for Evinacumab, a Treatment for HoFH, Receives Priority Review Status
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BLA for Evinacumab, a Treatment for HoFH, Receives Priority Review Status

  This week, Regeneron Pharmaceuticals announced that their Biologics License Application (BLA) for evanicumab, a treatment for homozygous familial hypercholesterolemia (HoFH), was granted Priority Review status by the FDA. The…

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Machine Learning Found to be a Better Diagnostic Tool than Dutch Lipid Score for Familial Hypercholesterolemia
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Machine Learning Found to be a Better Diagnostic Tool than Dutch Lipid Score for Familial Hypercholesterolemia

Ana Pina from the Centro de Estudos de Doencas Cronicas at NOVA Medical School in Portugal, and her colleagues recently published an innovative study regarding the diagnosis of familial hypercholesterolemia (FH).…

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Faulty Proteins and Failed Genes: Understanding the Biology Behind Congenital Diseases
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Faulty Proteins and Failed Genes: Understanding the Biology Behind Congenital Diseases

Patients with congenital diseases often experience a range of uncomfortable symptoms. On one side of the world, a family with erythromelalgia, a painful condition which can cause redness and swelling…

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Siblings with Familial Hypercholesterolemia to Receive Treatment at New Clinic
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Siblings with Familial Hypercholesterolemia to Receive Treatment at New Clinic

According to a story from The Scarborough News, brother Connor and sister Kiera Pickering, aged eleven and twelve years respectively, will be some of the first patients to be treated…

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Presentation Highlights Need for Improvements in Familial Hypercholesterolemia Care
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Presentation Highlights Need for Improvements in Familial Hypercholesterolemia Care

According to a story from Healio, a recent presentation featured at the FH Foundation Global Summit was a stark reminder of the need for improved treatment for patients with familial…

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Amgen is Utilizing Technology to Improve Diagnosis and Adherence in Rare Disease

Diagnosis, Treatment, and Adherence There are many factors necessary for an individual to be able to thrive with a serious medical diagnosis. First, they must receive the correct diagnosis so…

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This Drug Could be a Breakthrough for Severe Familial Hypercholesterolemia, But How Much Will it Cost?
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This Drug Could be a Breakthrough for Severe Familial Hypercholesterolemia, But How Much Will it Cost?

According to a story from Forbes, a new drug developed by the company Regeneron has the potential to make a real difference for people with the homozygous form of familial…

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When the Latest Treatments are Out of Reach, Familial Hypercholesterolemia Patients Suffer
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When the Latest Treatments are Out of Reach, Familial Hypercholesterolemia Patients Suffer

According to a story from FHM Pakistan, a recent study revealed the familial hypercholesterolemia patients are at a greater risk of stroke, heart attack, and other cardiovascular problems when they…

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Possible Treatment for Homozygous Familial Hypercholesterolemia Earns Orphan Drug Designation
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Possible Treatment for Homozygous Familial Hypercholesterolemia Earns Orphan Drug Designation

According to a story from bloomberg.com, the drug development company Arrowhead Pharmaceuticals, Inc. recently announced that its experimental product candidate AR0-ANG3 has earned Orphan Drug designation from the US Food…

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National Organization for Rare Disorders Announces Ten New Grants

In honor of its 30th anniversary, the National Organization for Rare Disorders (NORD) has announced ten new grants as part of its Rare Disease Research Grant Program! NORD is the…

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Could Blood Donations Improve Diagnostic Rates for Familial Hypercholesterolemia?
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Could Blood Donations Improve Diagnostic Rates for Familial Hypercholesterolemia?

According to a story from brightsurf.com, familial hypercholesterolemia, a genetic condition that causes elevated levels of LDL cholesterol to appear at a young age, often goes undiagnosed. In fact, some…

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Could This Revised Genetic Test Improve Diagnostic Rates for Familial Hypercholesterolemia?

According to a story from Healio, a recent study suggests that the addition of four more genes to the conventional genetic test used to diagnosed familial hypercholesterolemia could allow more…

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Artificial Intelligence(AI) and a Solution to Detect Familial Hypercholesterolemia

  Familial Hypercholesterolemia (FH) is often misdiagnosed as plain old high cholesterol because of overlapping symptoms such as elevated lipid levels. According to a recent article in MedicalView, FH is three…

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New Cholesterol-Lowering Drug Could Provide Alternative to Statins
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New Cholesterol-Lowering Drug Could Provide Alternative to Statins

According to a publication from EurekAlert, high-cholesterol patients, who do not take statins (a type of cholesterol-lowering medication often prescribed by doctors) due to adverse side effects may benefit from…

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Results From the Longest Familial Hypercholesterolemia Study to be Announced at the ACC 2019 Conference

New Research to be Presented In just a few days the American College of Cardiology will host their 68th Annual Scientific Session (ACC.19). It will be held in New Orleans,…

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Patient Centricity Helps Facilitate The Introduction of New Therapies for Rare Diseases
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Patient Centricity Helps Facilitate The Introduction of New Therapies for Rare Diseases

According to a story from Pharmaphorum, there are a number of new treatments that have been introduced or have seen major strides in development over the last year. More patient…

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2018 FH Global Summit Highlights

The 2018 FH Global Summit featured fantastic speakers, timely information, and valuable insight about the past, present and future of the way FH is understood, diagnosed, and managed. The summit…

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New Rare Diseases Have Been Added to the Patient Advocate Foundation’s Co-Pay Relief Program
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New Rare Diseases Have Been Added to the Patient Advocate Foundation’s Co-Pay Relief Program

The Patient Advocate Foundation has recently begun to expand the reach of its popular Co-Pay Relief program. The Co-Pay Relief program is designed to provide financial assistance to qualifying patients.…

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