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PASADENA, Calif.--(BUSINESS WIRE)--Arrowhead Pharmaceuticals, Inc. (NASDAQ: ARWR) today, on FCS Awareness Day, announced the launch of a new disease awareness campaign, 'We'll Get There Soon,' to bring hope to the rare disease community affected by Familial Chylomicronemia Syndrome (FCS). The campaign seeks to raise awareness about FCS, a severe and rare condition marked by persistently high triglyceride levels despite standard treatment, which can lead to serious and potentially life-threatening symptoms, including acute pancreatitis..
加利福尼亚州帕萨迪纳市(商业新闻短讯)--箭头制药公司(NASDAQ:ARWR)今天在FCS意识日宣布启动一项新的疾病意识活动,“我们很快就会到达”,为受家族性乳糜微粒血症综合征(FCS)影响的罕见疾病社区带来希望。该运动旨在提高人们对FCS的认识,FCS是一种严重而罕见的疾病,尽管经过标准治疗,其甘油三酯水平仍持续较高,可能导致严重且可能危及生命的症状,包括急性胰腺炎。。
The campaign features a dedicated website, www.LowerTriglycerides.com, along with social media pages on Instagram and Facebook offering a platform for people affected by FCS to share their experiences. A key focus is educating the community about expert guidelines, which recommend maintaining triglyceride levels below 500 mg/dL to reduce the risk of acute pancreatitis1-3..
该活动设有一个专门的网站www.LowerTriglycerides.com,以及Instagram和Facebook上的社交媒体页面,为受FCS影响的人提供了一个分享经验的平台。一个关键的重点是教育社区有关专家指南,该指南建议将甘油三酯水平维持在500 mg/dL以下,以降低急性胰腺炎的风险1-3。。
Arrowhead hosted a launch event at its Pasadena headquarters featuring special guests Julie Klueckman and her husband Gary, who shared their journey with FCS — both as a patient and a caregiver. Julie’s story highlights the significant challenges faced by those living with FCS, including multiple misdiagnoses, persistent and extended hospital stays, no available medicines that adequately treat FCS, and the necessity of maintaining an extremely low-fat diet of up to 20 grams per day — equivalent to less than three tablespoons of peanut butter..
Arrowhead在其帕萨迪纳总部举办了一场启动活动,特邀嘉宾朱莉·克鲁克曼(JulieKlueckman)和她的丈夫加里(Gary)作为患者和护理人员与FCS分享了他们的旅程。Julie的故事突显了FCS患者面临的重大挑战,包括多次误诊,持续住院和延长住院时间,没有足够治疗FCS的药物,以及必须保持每天高达20克的极低脂肪饮食-相当于不到三汤匙的花生酱。。
“My journey to diagnosis was a difficult one that ended after a decade-long diagnostic process and countless hours of research, learning about FCS, and finally presenting the idea to my doctor,” Julie explained. “Despite my eventful history with this condition, including countless hospitalizations, multiple surgeries, a radical hysterectomy, relocating and moving my family to be closer to my trusted doctors, I remain optimistic about the future for FCS patients.” Excited by what the future holds, Julie is also working with the Arrowhead team contributing patient insights to support and inform efforts for people with FCS because she believes, “Hope is on the horizon.”.
朱莉解释说:“经过长达十年的诊断过程和无数个小时的研究,学习了FCS,并最终向医生提出了这个想法,我的诊断之旅是一段艰难的旅程。”。“尽管我在这种情况下经历了无数次住院治疗,多次手术,根治性子宫切除术,重新安置和搬家,使我的家人更接近我信任的医生,但我仍然对FCS患者的未来感到乐观。”对未来的前景感到兴奋,Julie还与Arrowhead团队合作,为FCS患者提供患者见解,以支持和告知他们的努力,因为她相信“希望就在眼前”。
“We are humbled to play a small role in amplifying the voices of those affected by FCS and bringing them hope. Julie’s presence in our offices reminds us of why we do what we do every day and reaffirms one of Arrowhead’s core values: patients — making people’s lives better,” said Alexendra Roeser, Director of Patient Advocacy at Arrowhead..
Arrowhead患者倡导主任亚历克森德拉·罗瑟(AlexendraRoeser)表示:“我们很荣幸能在扩大受FCS影响者的声音并为他们带来希望方面发挥一点作用。朱莉出现在我们的办公室提醒我们为什么我们每天都在做什么,并重申了Arrowhead的核心价值观之一:患者-让人们的生活变得更好。”。。
Today, on FCS Awareness Day, and beyond, Arrowhead is on a mission to create awareness #ForFCS. Learn more at www.LowerTriglycerides.com.
今天,在FCS意识日及其后,Arrowhead的使命是为FCS创造意识。更多信息,请访问www.LowerTriglycerides.com。
About Familial Chylomicronemia Syndrome
关于家族性乳糜微粒血症综合征
Familial chylomicronemia syndrome (FCS) is a severe and rare genetic disease often caused by various monogenic mutations. FCS leads to extremely high triglyceride (TG) levels, typically over 880 mg/dL. Such severe elevations can lead to various serious signs and symptoms including acute and potentially fatal pancreatitis, chronic abdominal pain, diabetes, hepatic steatosis, and cognitive issues.
家族性乳糜微粒血症综合征(FCS)是一种严重而罕见的遗传疾病,通常由各种单基因突变引起。FCS导致甘油三酯(TG)水平极高,通常超过880 mg/dL。这种严重的升高可能导致各种严重的体征和症状,包括急性和潜在致命的胰腺炎,慢性腹痛,糖尿病,肝脂肪变性和认知问题。
Currently, there are no therapeutic options that can adequately treat FCS..
目前,没有可以充分治疗FCS的治疗选择。。
About Arrowhead Pharmaceuticals
关于箭头制药
Arrowhead Pharmaceuticals develops medicines that treat intractable diseases by silencing the genes that cause them. Using a broad portfolio of RNA chemistries and efficient modes of delivery, Arrowhead therapies trigger the RNA interference mechanism to induce rapid, deep, and durable knockdown of target genes.
箭头制药公司(Arrowhead Pharmaceuticals)通过沉默导致顽固性疾病的基因来开发治疗顽固性疾病的药物。使用广泛的RNA化学组合和有效的递送方式,箭头疗法触发RNA干扰机制,以诱导靶基因的快速,深入和持久的敲低。
RNA interference, or RNAi, is a mechanism present in living cells that inhibits the expression of a specific gene, thereby affecting the production of a specific protein. Arrowhead’s RNAi-based therapeutics leverage this natural pathway of gene silencing..
RNA干扰(RNAi)是活细胞中存在的一种机制,可抑制特定基因的表达,从而影响特定蛋白质的产生。Arrowhead基于RNAi的疗法利用了这种基因沉默的自然途径。。
For more information, please visit www.arrowheadpharma.com, or follow us on X (formerly Twitter) at @ArrowheadPharma, LinkedIn, Facebook, and Instagram. To be added to the Company's email list and receive news directly, please visit http://ir.arrowheadpharma.com/email-alerts.
欲了解更多信息,请访问www.arrowheadharma.com,或通过@arrowheadharma、LinkedIn、Facebook和Instagram在X(以前的Twitter)上关注我们。要添加到公司的电子邮件列表并直接接收新闻,请访问http://ir.arrowheadpharma.com/email-alerts.
Safe Harbor Statement under the Private Securities Litigation Reform Act:
私人证券诉讼改革法案下的安全港声明:
This news release contains forward-looking statements within the meaning of the 'safe harbor' provisions of the Private Securities Litigation Reform Act of 1995. Any statements contained in this release except for historical information may be deemed to be forward-looking statements. Without limiting the generality of the foregoing, words such as “may,” “will,” “expect,” “believe,” “anticipate,” “hope,” “intend,” “plan,” “project,” “could,” “estimate,” “continue,” “target,” “forecast” or “continue” or the negative of these words or other variations thereof or comparable terminology are intended to identify such forward-looking statements.
本新闻稿包含1995年《私人证券诉讼改革法案》中“安全港”条款所指的前瞻性声明。除历史信息外,本版本中包含的任何声明都可能被视为前瞻性声明。在不限制上述一般性的情况下,诸如“可能”、“将”、“预期”、“相信”、“预期”、“希望”、“打算”、“计划”、“项目”、“可能”、“估计”、“继续”、“目标”、“预测”或“继续”等词语或其其他变体或类似术语的否定词旨在识别此类前瞻性陈述。
In addition, any statements that refer to projections of our future financial performance, trends in our business, expectations for our product pipeline or product candidates, including anticipated regulatory submissions and clinical program results, prospects or benefits of our collaborations with other companies, or other characterizations of future events or circumstances are forward-looking statements.
此外,任何涉及我们未来财务业绩预测、业务趋势、对我们产品线或候选产品的期望(包括预期的监管提交和临床计划结果)、我们与其他公司合作的前景或益处,或未来事件或情况的其他特征的声明都是前瞻性声明。
These forward-looking statements include, but are not limited to, statements about the initiation, timing, progress and results of our preclinical studies and clinical trials, and our research and development programs; our expectations regarding the potential benefits of the partnership, licensing and/or collaboration arrangements and other strategic arrangements and transactions we have entered into or may enter into in the future; our beliefs and expectations regarding milestone, royalty or other payments that could be due to or from third parties under existing agreements; and our estimates regarding future revenues, research and development expenses, capital requirements and payments to third parties.
这些前瞻性陈述包括但不限于关于我们的临床前研究和临床试验以及我们的研究和开发计划的开始,时间,进展和结果的陈述;我们对合作伙伴关系、许可和/或合作安排以及我们已经达成或将来可能达成的其他战略安排和交易的潜在利益的期望;我们对里程碑、特许权使用费或现有协议下可能应付给第三方或来自第三方的其他付款的信念和期望;以及我们对未来收入、研发费用、资本要求和向第三方付款的估计。
These statements are based u.
这些声明基于美国。
Source: Arrowhead Pharmaceuticals, Inc.
来源:Arrowhead Pharmaceuticals,Inc。
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AACE/ACE Guidelines: Handelsman Y et al. Endocr Pract. 2020;26(10):1196-1224.
AACE/ACE指南:Handelsman Y等人,Endor Pract。2020年;。
ACC/AHA Guidelines: Grundy SM et al. Circulation. 2019;139(25):e1082-e1143.
ACC/AHA指南:Grundy SM等人。循环。2019年;139(25):e1082-e1143。
NLA Guidelines: Jacobson TA et al. J Clin Lipidol. 2015;9(2):129-169.
NLA指南:Jacobson TA等人,J Clin Lipidol。2015年;9(2):129-169。