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The new initiative aims to increase visibility and promote early detection of FH in communities of color on FH Awareness Day and during National Cholesterol Education Month WASHINGTON, Sept. 24, 2024 /PRNewswire/ -- The Association of Black Cardiologists (ABC) is proud to introduce its timely new campaign, "Family Heartbeats Unmasked - Sharing Stories. Empowering Communities. Saving Generations." This effort is dedicated to raising awareness about Familial Hypercholesterolemia (FH), a genetic condition that significantly increases the risk of early heart disease and is often underdiagnosed, especially in Black Americans and other minority populations. Association of Black Cardiologists' Family Heartbeats Unmasked Campaign - Christina's Story. "We are excited to share these moving stories and essential resources with our community," said Dr. Keith C. Ferdinand, Chief Science Officer of ABC. "Our goal is to educate and promote equitable care for individuals affected by FH, particularly in underserved populations." The campaign features powerful testimonials from individuals living with FH, like one of the ABC Patient Ambassadors who shares, "I wish I had known that it was familial at that time. That would really have helped me to understand a treatment plan and that it also is important to understand how it may impact my children as well." These stories underscore the importance of knowing your family health history and the life-changing impact of early detection for current and future generations. "Family Heartbeats Unmasked" highlights important stories on FH Awareness Day and during September's National Cholesterol Education Month. It also emphasizes the crucial significance of early diagnosis and treatment of FH, even in early childhood. FH affects approximately 1 in 250 people globally. Children with an affected parent have a 50% chance of inheriting FH, yet only about 30% of those born with the condition are diagnosed early. Up to 90% of individuals with FH remain undiagnosed, with Black Americans facing significant disparities in both timely identification and appropriate care. Through powerful patient stories, physician-led PSAs, and educational content, this initiative seeks to close the gaps in care and ensure that every individual, regardless of race/ethnicity, sex/gender, socioeconomic status, geography, or ability/disability, receives the care they need. The ABC is committed to equipping patients and healthcare professionals with the knowledge and tools necessary to improve outcomes for those living with FH. "It's especially important to recognize FH in children, as early detection through pediatric screening can be life-saving," said Dr. Ferdinand. "By bringing attention to FH and its impact on our communities, we hope to empower families with the tools they need to protect their heart health from the start." The ABC encourages everyone to visit the campaign's website at familyheartbeatsunmasked.org to watch the videos, access resources, and learn more about how to get involved. About the Association of Black Cardiologists (ABC) Founded in 1974, the Association of Black Cardiologists, Inc. (ABC) is a nonprofit with a global membership exceeding 2,000 individuals, including health professionals, community health advocates, and corporate and institutional members. The ABC remains dedicated to its mission of promoting heart health and wellness in minority communities and eliminating disparities in cardiovascular care for all people, with a particular focus on Black Americans. Through advocacy, education, and research, ABC works to ensure that every person has the opportunity to achieve optimal heart health. Today, ABC's public and private partnerships continue to make strides in improving cardiovascular outcomes in communities across the nation. Connect with us at and join our efforts on Twitter, Facebook, Instagram, and LinkedIn. SOURCE Association of Black Cardiologists WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

OXFORD, England, Sept. 19, 2024 /PRNewswire/ -- Research by Genomics plc has identified that 8% of the adult population have the same risk of heart disease due to polygenic risk, as carriers of rare genetic mutations that cause Familial hypercholesterolemia (FH). NHS testing currently focuses on the identification of monogenic conditions, such as Familial hypercholesterolemia, due to the increased cholesterol and heart disease risk caused by such conditions PRS measure the genetic component of risk for all the common diseases, including heart disease, diabetes, breast, and prostate cancer, with Genomics PRS outperforming all other competitors in all but a handful of diseases and traits PRS could reduce strain on healthcare systems by allowing them to focus on prevention and early detection of common disease, where the results of treatment are optimal Research describing best-practice polygenic risk scores (PRS) now published in a peer-reviewed journal A new paper published by Genomics plc in PLOS ONE indicates that a significant portion of the population (1 in 12) in the UK share the same pattern of risk of heart disease as FH mutation carriers. This is due to the cumulative effects of many genetic changes instead of monogenetic risk. Familial hypercholesterolemia (FH) is a genetic condition affecting 1 in 280 people, caused by a mutation in one of a few key genes that, if untreated, leads to high cholesterol levels and increased risk of heart disease. As a genetic condition, carriers of the mutation are often unaware. Substantial NHS resources are (rightly) spent on identifying individuals with this and similar rare genetic changes, as once identified, they can be treated with medication to reduce cholesterol and heart disease risk. Due to the impact of early intervention, the NHS's 10-year plan includes a key metric to improve the detection of FH individuals. Research indicated that the newly discovered polygenic group makes up 8% of the population and is responsible for 18 times more cases, than the FH group, of early heart disease (diagnosed before the age of 50). Even fewer people in this group are aware of their risk, but individuals can be identified via a simple test. Safe and inexpensive cholesterol lowering medications, such as statins, could then be prescribed to reduce risk for these individuals. Research has shown that statins are even more effective in this group, in lowering risk of heart attacks, than in the general population. The paper tested the performance of Genomics plc's polygenic risk scores, confirming the incomparable rigour of the company's approach to its genomic prevention technology. The PRS scores in the paper have been available for medical researchers to use in the UK Biobank for the last two years, with over 80 research papers already published using the Genomics risk scores. The study, which compares Genomics' PRS for 28 diseases and 25 traits against 76 other PRS, shows better performance for most diseases and traits. The paper quantifies the drop off in predictive power for individuals of non-European ancestry, but even in these other ancestries, PRS is useful for identifying high-risk individuals. Genomics' PRS also perform better in non-European ancestry individuals, in comparison to other PRS. Using their carefully tested PRS, the paper's researchers investigate the levels of risk currently unidentified in UK populations. PRS tools can play a critical role in identifying individuals at high risk of common diseases, but need to adhere to high standards of development, and performance assessment. Professor Sir Peter Donnelly FRS, FMedSci, Founder and Chief Executive Officer, Genomics plc commented: "It is untenable in the long term to offer interventions or enhanced screening to one group of individuals at high risk of heart disease because of genetics but not to another, just because the variants contributing to risk are different. It is an exciting landmark to have this paper published in PLOS ONE, as we continue to drive this area of medical research forward, given its very real world application. But while this is a pleasing validation of all our hard work, more importantly, we have found there is a much larger group of individuals with high PRS scores, who have the same pattern of risk for heart disease. We feel we have a crucial role to play in building awareness of this as we look to help people and the NHS." About Genomics plc Founded in 2014 by four world-leading statistical and human geneticists at the University of Oxford, Genomics plc (Genomics) is a healthcare company that uses large-scale genetic information to develop innovative precision healthcare tools and to bring new understanding to drug discovery to partners across the healthcare, insurance, employer and life sciences industries. Genomics collaborates with some of the world's leading organisations, helping them to predict, prevent, treat and cure disease using its proprietary algorithms and databases – reducing the human and financial cost of critical diseases like cancer and diabetes. For additional information about Genomics, please visit or on LinkedIn or Twitter. Media contact: [email protected] SOURCE Genomics plc WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

Evkeeza is a first-in-class medicine approved by Health Canada, the U.S. Food and Drug Administration (FDA) and European Commission (EC) to treat an ultrarare, inherited form of high cholesterolTORONTO, Sept. 25, 2023 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development and commercialization of novel therapies for rare and ultrarare genetic diseases, today announced the approval of Evkeeza® (evinacumab) in Canada. Evkeeza® was approved by Health Canada as an adjunct to diet and other low-density lipoprotein cholesterol (LDL-C) lowering therapies for the treatment of adult and pediatric patients aged 5 years and older with homozygous familial hypercholesterolemia (HoFH) and will be commercially available in Canada effective 25-November-2023. Evkeeza is a first-of-its-kind treatment for HoFH and is the first monoclonal antibody inhibiting the angiopoietin-like 3 protein (ANGPTL3). Evkeeza is delivered via 60-minute intravenous infusion every 4 weeks. The treatment is now available to prescribe for patients with HoFH in Canada. “The approval and launch of Evkeeza in Canada for the treatment of homozygous familial hypercholesterolemia exemplifies our commitment to bring innovative therapies for people living with rare and ultrarare genetic diseases,” said Monty Keast, Vice President and General Manager at Ultragenyx Canada. “We look forward to working collaboratively with health care providers, the HoFH patient community and payers from across the country to make this potentially life-changing therapy accessible to people living with this serious disease.” “HoFH is a severe atherosclerotic disease associated with premature cardiovascular morbidity, which most often does not adequately respond to cholesterol-lowering treatment currently available,” said Prof. Daniel Gaudet, Lipidologist and Director of the Severe Lipid Disorders Unit at the Community Genetic Medicine Center, Department of Medicine, Université de Montréal. “Our experience in recent years in phase 2 and 3 clinical studies, clearly demonstrates that in combination with diet and other low-density lipoprotein-cholesterol (LDL-C) lowering therapies, evinacumab represents an effective option to substantially lower the levels of LDL-cholesterol and other atherogenic lipoproteins in HoFH patients.” About Homozygous Familial Hypercholesterolemia (HoFH) HoFH, also known as homozygous FH, is the most severe form of inherited hypercholesterolemia. This ultrarare disease affects 1 in 300,000 people worldwide. There is a founder effect in the French-Canadian population with a prevalence estimated to be 1 in 250,000 people. Homozygous familial hypercholesterolemia runs in families and is usually passed down by both father and mother. People with this condition have extremely high levels of LDL-cholesterol (‘bad cholesterol’) from birth. Such high levels can lead to heart attacks, heart valve disease, or other problems at an early age. For more information about HoFH please go to www.hofhdisease.ca. About Evkeeza® (evinacumab) Evinacumab, the active substance in Evkeeza, attaches to a protein in the body called ANGPTL3 and blocks its effects. ANGPTL3 is involved in controlling cholesterol levels and blocking its effect reduces the level of cholesterol in the blood. Evkeeza is delivered via an infusion every 4 weeks. Evkeeza is approved by Health Canada as an adjunct to diet and other low-density lipoprotein cholesterol (LDL-C) lowering therapies for the treatment of adult and pediatric patients aged 5 years and older with homozygous familial hypercholesterolemia (HoFH). The effects of Evkeeza on cardiovascular morbidity and mortality have not been determined. Regeneron Pharmaceuticals, Inc. (NASDAQ: REGN) discovered and developed Evkeeza, and solely commercializes the product in the U.S. Ultragenyx is responsible for commercialization efforts for Evkeeza in countries outside of the U.S. Indication In Canada, EVKEEZA® (evinacumab for injection) is indicated as an adjunct to diet and other low-density lipoprotein cholesterol (LDL-C) lowering therapies for the treatment of adult and pediatric patients aged 5 years and older with homozygous familial hypercholesterolemia (HoFH). The effects of Evkeeza on cardiovascular morbidity and mortality have not been determined. IMPORTANT SAFETY INFORMATION FOR EVKEEZA® (evinacumab) INFUSION To help avoid side effects and ensure proper use, talk to your healthcare professional before you take Evkeeza. Hypersensitivity reactions, including anaphylaxis and infusion reactions (e.g., infusion site pruritus), have been reported with Evkeeza. If signs or symptoms of serious hypersensitivity reactions occur, discontinue treatment with Evkeeza, treat according to the standard of care, and monitor until signs and symptoms resolve. The most common adverse reactions to Evkeeza were nasopharyngitis (13.7% vs. 13.0% in placebo), influenzalike illness (7.7% vs. 5.6% in placebo), dizziness (6.0% vs. 0% in placebo), back pain (5.1% vs. 3.7% in placebo), and nausea (5.1% vs. 1.9% in placebo). Fatigue (15%) was identified as an adverse reaction to Evkeeza for pediatric patients aged ≥ 5 to 11 years only. Who should not use Evkeeza? Evkeeza may not be used if you are allergic to evinacumab or to any of the ingredients in Evkeeza. If you are pregnant, think you might be pregnant, or plan to become pregnant, ask your healthcare professional for advice before taking Evkeeza. Evkeeza may harm your unborn baby. Tell your healthcare professional if you become pregnant while using Evkeeza. For people who are able to become pregnant: Your healthcare professional may do a pregnancy test before you start treatment with Evkeeza.You should use an effective method of birth control during treatment and for at least 5 months after the last dose of Evkeeza. Talk to your healthcare professional about birth control methods that you can use during this time.If you are breastfeeding or plan to breastfeed, ask your healthcare professional for advice before you are given Evkeeza. It is not known if Evkeeza passes into your breast milk. You and your healthcare professional should decide if you will receive Evkeeza or breastfeed. You may report side effects via email to ultragenyx@primevigilance.com. Please see PRODUCT MONOGRAPH INCLUDING PATIENT MEDICATION INFORMATION for more information. About Ultragenyx Pharmaceutical Inc. Ultragenyx is a biopharmaceutical company committed to bringing novel products to patients for the treatment of serious rare and ultrarare genetic diseases. The company has built a diverse portfolio of approved therapies and product candidates aimed at addressing diseases with high unmet medical need and clear biology for treatment, for which there are typically no approved therapies treating the underlying disease. The company is led by a management team experienced in the development and commercialization of rare disease therapeutics. Ultragenyx’s strategy is predicated upon time- and cost-efficient drug development, with the goal of delivering safe and effective therapies to patients with the utmost urgency. For more information on Ultragenyx, please visit ultragenyx.ca. Contacts Ultragenyx Pharmaceutical, Inc.MediaJeff Blake+1-415-612-7784media@ultragenyx.com