Developmental Disabilities

Country’s Most Experienced Long-term Care Accountable Care Organization Shares $44.1 Million in Savings with Participating Providers Committed to Improving Quality and Cost of CareKENNETT SQUARE, Pa., Oct. 30, 2024 (GLOBE NEWSWIRE) -- LTC ACO, the first Accountable Care Organization (ACO) in the U.S. dedicated solely to long-term care facility residents, today announced it received a positive reconciliation and settlement under the Medicare Shared Savings Program (MSSP) for the 2023 performance year. As LTC ACO celebrates a decade in the MSSP, it anticipates significant growth, including expanding its services to assisted living facilities and continuing to lead in enhancing care for high-need populations. "We are extremely proud to announce that LTC ACO and our participating practices have once again achieved shared savings under the CMS Medicare Shared Savings Program, generating one of the highest savings rates on a per beneficiary basis in the country," said Jason Feuerman, president and CEO of LTC ACO. “Our positive results are a testament to the commitment of our participating providers, who work tirelessly to enhance care for the Medicare population living in long-term care facilities. We are grateful for the trust they place in us as their value-based care partner.” In 2023, LTC ACO attributed more than 16,000 Medicare fee-for-service beneficiaries to its MSSP ACO. The organization achieved total savings to CMS of nearly $60 million, resulting in earned shared savings of $44.1 million to LTC ACO. Since 2018, LTC ACO has saved Medicare more than $132 million demonstrating its commitment to cost-effective, high-quality care for long-term care facility residents. “As we enter our 10th year of MSSP participation, our mission remains focused on enabling the success of our partners in value-based care by providing innovative and effective insights and interventions,” said Feuerman. “Together with our partners, we will continue transforming healthcare quality for long-stay facility residents through industry-leading, value-based care solutions.” Leveraging its extensive experience, LTC ACO supports the value-based care success of providers serving patients in long-stay facilities. As an early adopter of CMS’s new quality reporting framework, LTC ACO continues to set higher standards in long-term care, pioneering advancements in health outcomes and reducing costs for long-term care residents. By leveraging innovative approaches, such as artificial intelligence for predictive analytics, LTC ACO enhances clinical analysis and reporting, helping our practices focus on their patients at greatest risk for avoidable hospitalizations to improve outcomes for its unique population of beneficiaries. LTC ACO focuses on a high need, underserved subset of the Medicare population. This includes the frail elderly, as well as individuals living with Alzheimer’s disease or dementia, depression, amputations, developmental or physical disabilities, renal failure and those with multiple chronic diseases and complications who reside in long-stay facilities. Over 85% of LTC ACO’s population is “dually” eligible, participating in Medicare and Medicaid. As the most experienced MSSP ACO serving practices treating these beneficiaries today, LTC ACO creates reliable shared savings revenue opportunities with no downside risk for its participating practices. To learn more about how LTC ACO helps providers and facilities achieve financial success and improve clinical impact in Medicare’s value-based care programs, visit http://www.ltcaco.com. About LTC ACOLTC ACO is the first Accountable Care Organization (ACO) in the United States dedicated to long-term care facility residents. LTC ACO began participating in the Medicare Shared Savings Program (MSSP) in 2016 for Medicare beneficiaries who reside in long-stay facilities. Visit http://www.ltcaco.com to explore opportunities for participation. Media Contact:Nerida Brennan610-925-1001

Wholly owned first-in-class program anticipated to enter clinical trials in first half of 2025 THOUSAND OAKS, Calif., Oct. 30, 2024 /PRNewswire/ -- Capsida Biotherapeutics ("Capsida") today announced the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) to Capsida for CAP-002. CAP-002 is the company's lead investigational gene therapy for the treatment of developmental and epileptic encephalopathy (DEE) due to syntaxin-binding protein 1 (STXBP1) mutations. CAP-002 is a novel, first-in-class IV-administered gene therapy using one of Capsida's proprietary engineered capsids designed to achieve brain-wide neuronal expression of the STXBP1 protein while significantly detargeting the liver. CAP-002 is currently in IND-enabling studies. "FDA's granting Orphan Drug Designation signals the significant potential that CAP-002 demonstrates based upon our preclinical data," said Peter Anastasiou, Chief Executive Officer of Capsida Biotherapeutics. "There are no disease-modifying therapies available for STXBP1 developmental and epileptic encephalopathy and CAP-002 could be the first, bringing hope for patients and families affected by this devastating disorder." The STXBP1 protein is present in every neuron in the brain and is essential for normal neurotransmission. A mutation in the STXBP1 gene is associated with treatment-resistant seizures, severe developmental delay and intellectual disability, motor abnormalities, and sudden unexpected death in epilepsy (SUDEP). Preclinical pharmacology studies have established the potential to treat and even fully correct the disease through gene supplementation. These studies demonstrated rescue of these neurological phenotypes was dependent on supplementation of STXBP1 in neurons throughout the brain at levels not achievable by wild-type serotypes, such as AAV9. Capsida conducted these studies with a proprietary murine disease model in collaboration with Mingshan Xue, Ph.D., associate professor of neuroscience and molecular and human genetics at Baylor College of Medicine, as well as a member of the Cain Foundation Laboratories and the Duncan Neurological Research Institute at Texas Children's Hospital. "Receiving Orphan Drug Designation moves us one step closer to our goal of transforming the lives of people living with STXBP1 developmental and epileptic encephalopathy," said Swati Tole, M.D., Chief Medical Officer at Capsida. "We are committed to advancing CAP-002, with our IND filing in the first half of 2025, and bringing novel gene therapies to people with high unmet medical needs." The FDA's Orphan Drug Designation is intended to encourage the development of treatments for rare diseases, defined as those affecting fewer than 200,000 people in the United States. Orphan Drug Designation may shorten the clinical development path through closer FDA collaboration and potential qualification for expedited development programs. This designation also provides the company with certain benefits, including tax credits for qualified clinical trials, exemption from user fees, and potential seven-year market exclusivity upon FDA approval. About STXBP1 Developmental and Epileptic Encephalopathy Developmental and epileptic encephalopathy caused by STXBP1 mutations is estimated to affect up to one in 26,000 children globally. It is associated with treatment resistant seizures, severe developmental delay and intellectual disability, motor abnormalities, and sudden unexpected death in epilepsy (SUDEP). There are no approved treatments. About Capsida Biotherapeutics Capsida Biotherapeutics is a fully integrated gene therapy company with a central nervous system (CNS) pipeline consisting of disease modifying and potentially curative treatments for rare and more common diseases across all ages. Capsida's intravenously (IV) administered gene therapies utilize proprietary engineered capsids that enable high transduction levels to desired tissues and cells, while limiting tropism to non-target organs, such as the liver. Capsida has three wholly owned programs, including a potential -first-in-class treatment for STXBP1 developmental and epileptic encephalopathy and a best-in-class treatment Parkinson's disease associated with GBA mutations, both of which are in IND-enabling studies and on track to enter the clinic in the first half of 2025. In addition to its wholly owned programs, the Company has validating CNS partnerships with AbbVie, Lilly, CRISPR Therapeutics, and the AbbVie partnership was expanded to include ophthalmology disorders. Capsida was founded in 2019 by lead investors Versant Ventures and Westlake Village BioPartners and originated from groundbreaking research in the laboratory of Viviana Gradinaru, Ph.D., a neuroscience professor at Caltech. Visit us at . SOURCE Capsida Biotherapeutics WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

10月24日至27日，由上海市医学会、上海市医学会脑卒中专科分会主办的东方脑血管病会议2024（OCIN 2024）暨上海市医学会脑卒中专科分会学术年会在上海国际会议中心隆重召开。本届大会汇聚国内外众多专家学者和业界精英，分享脑血管病诊疗新技术、新理念。 本次大会上，来自武汉大学中南医院的梅斌教授对“脑静脉窦狭窄&血栓诊疗进展“进行详解，本文梳理重点内容，以分享各位同道。 01 脑静脉窦简介 脑静脉窦位于两层硬脑膜之间，壁内有很多毛细血管和小动脉，窦的内面有一层扁平细胞，是和静脉相连的内皮细胞。各静脉窦之间相互连通。Durgun等在1993年就已经通过造影证实，正常人群中只有 37.6% 的人在脑静脉窦回流通路中的双侧横窦和乙状窦是对称的，一般情况下把主要承担来自矢状窦和窦汇回流血液任务的一侧横窦和乙状窦称之为优势引流侧静脉窦，另一侧为非优势引流侧。 脑静脉窦病变包括脑静脉窦狭窄，脑静脉窦血栓以及静脉窦发育不良。脑静脉窦狭窄是一个形态学的影像诊断，其原因可能是蛛网膜颗粒、血栓或外在压迫。这种形态学诊断可能会有临床意义（颅高压等），也可能无临床意义。脑静脉窦血栓是一个病理学诊断，可导致静脉窦狭窄或闭塞。静脉窦发育不良以一侧横窦发育不良最常见，仅为变异，非疾病，在特定情况下有临床意义。 Marmarou 等和 Stevens等研究认为，一般静脉窦狭窄处管径较正常管径缩窄 40% 以上可诊断静脉窦狭窄，而若管径较对侧缩窄 40% 以上，则考虑为静脉窦发育不全。在影像学诊断上，由于非优势引流一侧横窦常因血流减少而显影较差或者不显影，常被误认为存在横窦狭窄或者闭塞。 02 脑静脉窦狭窄 脑静脉窦狭窄（VSS）是一种少见的脑血管疾病，每年发病率为1-2/100000，常见病因包括： 内源性：静脉窦壁畸形（蛛网膜颗粒、瘢痕组织等)、静脉窦壁解剖变异(静脉分隔、盲袋结构)外源性：颅内占位、脑脊液压力升高 脑静脉窦狭窄的临床症状为静脉窦狭窄若发生于优势一侧静脉窦可出现头痛，严重时可以导致静脉回流受阻，进而引起脑脊液的吸收障碍及颅内压升高，表现为头痛、视力下降等。脑静脉窦狭窄因血液湍流还可以出现搏动性耳鸣（PT）等症状。 脑静脉窦狭窄导致颅内压升高的机制: ①静脉窦狭窄引起脑静脉回流障碍，脑静脉压力升高导致颅内压升高； ②静脉窦狭窄导致上矢状窦内静脉压升高，再引起上矢状窦内蛛网膜颗粒吸收脑脊液减慢，从而导致颅内压升高，最后升高的颅内压又会压迫静脉窦，导致静脉窦的进一步狭窄，从而形成恶性循环。 影像学评估方法---DSA 1、DSA是诊断脑静脉窦狭窄的金标准 由于DSA是一种有创性检查，单纯诊断静脉窦狭窄较少直接进行 DSA 检查。必须采用DSA的情况： ①通过其他手段无法明确静脉窦狭窄诊断；②需要排除硬脑膜动静脉痿等其他疾病时； DSA 检查+静脉窦内逆行造影+微导管测压术可更进一步判断静脉窦狭窄部位狭窄程度及窦内压力变化，有助于更准确了解静脉窦狭窄的程度和血流动力学状况，并判断患者是否适合介入手术。然而，DSA不足之处是不能提供静脉窦血管壁和近结构的解剖信息，不利于提供颅内静脉窦狭窄的原因。 2、脑静脉窦狭窄的新型诊断方法 Farb 等通过改进的自动触发螺旋型填充3D增强 MRV（ATECOMRV）分析上矢状窦-横窦-乙状窦影像，对左、右横窦及乙状窦的狭窄程度进行评分分级，结论认为ATECOMRV 的成像质量与 DSA 3D重建后图像类似，该方法尚有待于进一步通过大样本证实。 动态 4D-MR 血管成像技术是一种动态评估手段，可以隐藏静脉前的动静脉分流影像从而完全显示个颅内静脉网，可分析静脉窦狭窄后血流动力学变化，特别是可显示狭窄附近形成的涡流，对静脉窦相关性耳鸣的诊断有重要意义。 静脉血管内超声(IVUS)可以提供静脉窦内腔的 360°视图，并可同时显示静脉周围解剖结构，且无需对比剂。IVUS 可以和颅内静脉压测量同时进行，可精确测量狭窄的程度和长度，以及狭窄类型(内生型狭鹕踪窄和外压型狭窄)。介入治疗后，IVUS 还可用来评估治疗效果、监测再狭窄等(包括准确定位狭窄位置、描述狭窄部位形态学特征、鉴别支架内血栓)。 因此，术前充分完善静脉窦狭窄相关无创影像学评估(包括 CT、MRI、CTV、MRV、MR BTI等），初步判断是否存在静脉窦狭窄，并结合多种影像学手段评估静脉窦狭窄段的形态、长度、部位、以及静脉侧支循环代偿状态。高度怀疑静脉窦狭窄后，建议进行DSA检查，明确静脉窦狭窄情况，并建议同期进行静脉窦内逆行造影及微导管测压术等，明确狭窄程度及狭窄段远近端压力差。 几种新技术（自动触发螺旋型填充3D增强 MRV、动态 4D-MR 血管成像技术、静脉血管内超声）仍缺乏大样本支持，其准确性及有效性有待于进一步评估。 3、脑静脉窦狭窄的介入治疗方法 症状性静脉窦狭窄主要的介入治疗方法为脑静脉窦支架置入术。与动脉病变不同的是，由于窦壁的弹性回缩及静脉血管内没有使之保持通畅的高压血流，因此静脉窦狭窄极少对单纯球囊扩张有效，只有支架可保证静脉窦的持续畅通。 由于脑静脉窦管径较大，需要有一定径向支撑力且直径较大的血管支架系统，但颈静脉孔区的骨嵴结构难以通过较硬的外周支架输送系统，因此增加了介入手术的难度，使脑静脉窦支架技术的推广较为缓慢。 03 脑静脉窦血栓 脑静脉窦血栓形成(CVT)约90%患者可出现头痛，颅高压20%~40% 患者可出现癫痫，20%~50% 出现局灶性神经功能缺损症状。CVT 的发生部位包括上矢状窦约占 25%~45%，直窦约 15%~18%，横窦约 25%~60%，皮层静脉约 15%~17%。CVT易感因素包括性别、激素水平、肿瘤、免疫、遗传等。 1、CVT动物模型的建立 Virchow血栓形成三要素包括改变血流状态、损害上矢状窦内皮、引起血液高凝状态。截至目前，在通过实验模型复制 CVT 的所有阶段，实现符合临床特征的转化研究仍然具有挑战。 2、CVT影像学评估方法 CT及 MR 是首选的诊断手段，可显示：①血栓直接征象②静脉流空影消失③静脉回流受阻继发改变（静脉性梗死、水肿、出血转化、颅高压、脑积水等） 怀疑皮质静脉血栓时推荐 SWI、MRBTI 和T2*GRE序列； 对慢性血栓，推荐增强 MRBTI 及 4D CE-MRV序列； 2，CVT治疗 ①抗凝治疗 抗凝治疗是 CVT 治疗的核心手段。起始一般选择皮下注射低分子肝素，随后过渡为维生素 K拮抗剂（如华法林）口服3~12月，服药具体时间根据其病因及易感因素确定。 ②血管内治疗 现阶段，EVT 主要作为 CVT 患者临床功能恶化时的补救治疗措施，并非首选方案。 ③去骨瓣减压 去骨瓣减压用于重症CVT和即将发生脑疝的实质病变的患者。DECOMPRESS2 研究，一项在欧洲、亚洲和美洲10个国家15个中心进行的前瞻性多中心队列研究，收纳了118名接受去骨瓣减压的CVT患者，旨在评估12个月死亡和重度残疾(mRS 5-6)，次要结局为出院时死亡，6个月和12个月死亡、功能完全恢复(mRS 0-1)，功能独立(mRS 0-2)，严重残疾(mRS 4-5)。结果显示，研究发现近2/3的CVT患者在术后一年仍存活，超过1/3的患者能够独立生活，该结果为指南推荐的去骨瓣减压术提供了新的证据。 来源 | 梅斯医学官网 编辑 | Swagpp ● 持续热议！广东一公立医院欠薪数月，停诊破产后，医生：头次听说公立也会倒闭，我们这也欠薪，会步入后尘吗？二级医院艰难处境受到关注 ● 复旦大学惊人发现：仅3天的厨房烹饪油烟，就能引起肺炎和损伤肠道！持续7天更严重 ● 重磅！糖尿病是SGLT2抑制剂的禁忌症？第十版内科学出现错误，主编葛均波院士亲自回应！积极给教材挑错，本科教育要细之又细！ 版权说明：梅斯医学（MedSci）是国内领先的医学科研与学术服务平台，致力于医疗质量的改进，为临床实践提供智慧、精准的决策支持，让医生与患者受益。欢迎个人转发至朋友圈，谢绝媒体或机构未经授权以任何形式转载至其他平台。 点击下方「阅读原文」 立刻下载梅斯医学APP！