更新于：2024-11-01

Hypothalamic Diseases

下丘脑疾病

更新于：2024-11-01

基本信息

别名

Disease, Hypothalamic、Diseases, Hypothalamic、Disorder of hypothalamus

+ [24]

简介

Neoplastic, inflammatory, infectious, and other diseases of the hypothalamus. Clinical manifestations include appetite disorders; AUTONOMIC NERVOUS SYSTEM DISEASES; SLEEP DISORDERS; behavioral symptoms related to dysfunction of the LIMBIC SYSTEM; and neuroendocrine disorders.

关联

229

聚乙二醇重组人生长激素 (长春金赛)

靶点

GHR

作用机制

GHR激动剂

在研机构

长春金赛药业有限责任公司

原研机构

长春金赛药业有限责任公司

在研适应症

生长激素缺乏症 [+5]

非在研适应症-

最高研发阶段批准上市

首次获批国家/地区

中国

首次获批日期2024-08-13

Pafolacianine

靶点

FOLR1 x FOLR2

作用机制

FOLR1激动剂 [+2]

在研机构

University of California, Irvine [+1]

原研机构

On Target Laboratories LLC

在研适应症

肺癌 [+5]

非在研适应症

子宫内膜癌 [+5]

最高研发阶段批准上市

首次获批国家/地区

美国

首次获批日期2021-11-29

Somatrogon(OPKO Health, Inc.)

靶点

GHR

作用机制

GHR激动剂

在研机构

OPKO Health, Inc. [+7]

原研机构

OPKO Health, Inc.

在研适应症

侏儒症 [+2]

非在研适应症-

最高研发阶段批准上市

首次获批国家/地区

加拿大

首次获批日期2021-10-26

1,397

项与下丘脑疾病相关的临床试验

NCT05645211 / Not yet recruitingN/AIIT

Agouti-related Peptide (AgRP) and the GH/IGF-1 Axis in Children

Recent data support the existence of a GH-Agouti-related peptide (AgRP) axis. The neuropeptide AgRP promotes food intake and has important effects on energy homeostasis. Recent evidence suggest that GH stimulates AgRP and AgRP may mediate some of GH's important nutritional and metabolic effects. main goals of this project are to characterize, for the first time, plasma levels of AgRP in children and to determine how these relate to GH and IGF-1 levels, age, body composition, clinical and other endocrine parameters. To accomplish this, we will conduct two studies, one being a cross-sectional study that will measure AgRP levels in 140 healthy children ages 5-17 and the second being a prospective study that will measure the change in plasma AgRP levels in response to GH treatment in 16 children who receive this as part of their clinical care for GH deficiency or short stature.

开始日期2025-06-10

申办/合作机构

Columbia University

NCT06368817 / Recruiting临床2期IIT

A Phase II Trial Evaluating Chemotherapy Followed by Response-Based Reduced Radiation Therapy for Patients With Central Nervous System Germinomas

This phase II trial studies how well lower dose radiotherapy after chemotherapy works in treating children with central nervous system (CNS) germinomas. Radiation therapy uses high energy x-rays, particles, or radioactive seeds to kill cancer cells and shrink tumors. Carboplatin is in a class of medications known as platinum-containing compounds. It works in a way similar to the anticancer drug cisplatin, but may be better tolerated than cisplatin. Carboplatin works by killing, stopping or slowing the growth of tumor cells. Etoposide is in a class of medications known as podophyllotoxin derivatives. It blocks a certain enzyme needed for cell division and DNA repair and may kill cancer cells. Researchers want to see if lowering the dose of standard radiotherapy (RT) after chemotherapy can help get rid of CNS germinomas with fewer long-term side effects.

开始日期2025-06-06

申办/合作机构

The Children's Oncology Group Foundation, Inc.

NCT04867317 / Not yet recruiting临床3期IIT

CSP #2018 - Growth Hormone Replacement Therapy in Veterans With Mild Traumatic Brain Injury (mTBI) and Adult Growth Hormone Deficiency (AGHD)

The purpose of this study is to determine whether growth hormone replacement therapy (GHRT) is effective versus placebo in the improvement of Quality of Life in patients with adult growth hormone deficiency (AGHD) and mild traumatic brain injury (mTBI).

开始日期2025-01-01

申办/合作机构

VA Office of Research and Development

100 项与下丘脑疾病相关的临床结果

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100 项与下丘脑疾病相关的转化医学

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0 项与下丘脑疾病相关的专利（医药）

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91,138

项与下丘脑疾病相关的文献（医药）

2025-04-01·Archivos Argentinos de Pediatria

Diencephalic syndrome in a child with chronic malnutrition

Article

作者: Rostagno, Gonzalo ; Rodriguez Schulz, Diego ; Dingevan Cassab, Jessica

Russell's diencephalic syndrome is a set of signs and symptoms characterized by extreme weight loss, with no impairment of height or head circumference, without changes in intake or appetite. It is due to hypothalamic dysfunction associated with space-occupying lesions in this region. It is a rare cause of malnutrition in pediatrics, so its diagnosis is often delayed. Here we describe the case of a 17-month-old male patient with weight loss for 3 months who was admitted to the pediatric inpatient ward due to suspected intestinal malabsorption syndrome. After ruling out the most common causes of malnutrition, the patient was diagnosed with pilomyxoid astrocytoma of the brainstem.

2025-01-01·The Journal of Pediatrics

Maternal Diabetes and Neurodevelopmental Outcomes of Infants Born Before 29 Weeks’ Gestation

Article

作者: Khurshid, Faiza ; Cieslak, Zenon ; Reichert, Amber ; Strueby, Lannae ; Barrington, Keith ; St-Hilaire, Marie ; Ricci, M. Florencia ; Lee, Kyong-Soon ; Moddemann, Diane ; Kanungo, Jaideep ; Zwicker, Jill ; Sherlock, Rebecca ; Shah, Prakesh S. ; Ng, Eugene ; Emberley, Julie ; Lapoint, Anie ; Bizgu, Victoria ; Beltempo, Marc ; Moodley, Anitha ; Canning, Roderick ; Afifi, Jehier ; Mandel, Ruth ; Coughlin, Kevin ; Fajardo, Carlos ; Banihani, Rudaina ; Church, Paige ; Lee, Shoo K. ; Monterrosa, Luis ; Hendson, Leonora ; Kelly, Edmond ; Mehrem, Ayman Abou ; Morin, Alyssa ; Akinseye, Adetokunbo ; Piedboeuf, Bruno ; Mukerji, Amit ; Drolet, Christine ; Seshia, Mary ; Makary, Hala ; Daboval, Thierry ; Ricci, Florencia ; de Cabo, Cecilia ; Hicks, Matthew ; Nguyen, Kim-Anh ; Lemyre, Brigitte ; Stavel, Miroslav ; McKnight, Sarah ; Ojah, Cecil ; Aziz, Khalid ; Alvaro, Ruben ; Augustine, Sajit ; Pillay, Thevanisha ; Belanger, Sylvie ; Pylypjuk, Christy ; Synnes, Anne ; Da Silva, Orlando ; Wang, Dianna ; strueby, Lannae ; Ting, Joseph ; Louis, Deepak ; Bodani, Jaya ; Ly, Linh ; Toye, Jennifer ; Kajetanowicz, Andrzej ; Claveau, Martine

OBJECTIVETo compare the neurodevelopmental outcomes of infants born at <29 weeks' gestation and exposed to diabetes in pregnancy with those unexposed.STUDY DESIGNThis was a retrospective cohort study using the Canadian Neonatal Network and Canadian Neonatal Follow-Up Network databases. Infants born <29 weeks' gestation and admitted to a level 3 neonatal intensive care unit from 2009 through 2018 who had neurodevelopmental assessments at 18-24 months corrected age were eligible. The 2 primary outcomes were as follows: (1) Neurodevelopmental impairment (NDI) (≥1 of Bayley-III scores < 85 in any domain, cerebral palsy, or vision or hearing impairment); and (2) significant NDI (sNDI) (≥1 of Bayley-III scores < 70 in any domain, cerebral palsy Gross Motor Function Classification System ≥ 3, bilateral blindness, or need for hearing aids or cochlear implants). Secondary outcomes were the individual components of NDI and sNDI. Adjusted odds ratios with 95% CIs were calculated to determine outcomes between groups.RESULTSOf 13 988 eligible infants, 55% attended neurodevelopmental follow-up assessments. Infants exposed to diabetes had increased odds of NDI compared with those unexposed (aOR 1.09 (95% CI 1.08-1.54); there was no difference in sNDI (aOR 1.07 (95% CI 0.84-1.36). Language and motor delays were more common in those exposed to maternal diabetes.CONCLUSIONSHigher rates of NDI, language, or motor delays were present in infants born at <29 weeks' gestation exposed to diabetes in utero. Future research is needed to determine the etiology and clinical significance of these findings.

2025-01-01·Biochimica et Biophysica Acta (BBA) - Molecular Basis of Disease

Conditional Pten inactivation in pituitary results in sex-specific prolactinoma formation

Article

作者: Soto-Moreno, Alfonso ; Barroso, Alexia ; Martínez-Barberá, Juan Pedro ; Ramos-Herrero, Víctor Darío ; Cano, David A. ; G-García, Miguel E ; Flores-Martínez, Álvaro ; Moreno, Alicia ; Cano, David A ; Dios, Elena ; Luque, Raúl M. ; Luque, Raúl M ; G-García, Miguel E. ; Venegas-Moreno, Eva

Pituitary tumors, including prolactinomas, present significant clinical challenges that require a deeper understanding of their molecular roots for improved diagnostics and therapies. Here, we investigate the role of the phosphatase and tensin homolog (PTEN)/phosphoinositide 3-kinase (PI3K) pathway in pituitary tumorigenesis using a mouse model. Conditional knockout of Pten in all pituitary cell lineages resulted in prolactinoma formation exclusively in female mice, demonstrating the critical role of PTEN in pituitary homeostasis. While Pten inactivation induced Akt activation in all pituitary cells, only prolactin-producing cells exhibited tumorigenic changes, suggesting specific cell-type effects. Histological and molecular analyses of prolactinomas revealed similarities with human pituitary tumors, such as decreased vascularization and cell adhesion proteins and increased accumulation of cell cycle proteins. Notably, prolactinomas displayed diminished levels of phosphorylated extracellular signal-regulated kinase (ERK), implicating downregulation of ERK in tumorigenesis. Finally, we analyzed PTEN/PI3K activation in a collection of human pituitary tumors. Overall, our study delineates the intricate interplay between the PTEN and ERK signaling pathways, providing insights into sex-specific mechanisms of pituitary tumorigenesis and potential therapeutic strategies for prolactinomas.

1,525

项与下丘脑疾病相关的新闻（医药）

2024-10-31

·药事纵横

缓释微球制剂优势、挑战及应用发展！

点击图片查看详情 · 海口亚特(国际)会议中心酒店 *如何领取手册-添加秘书微信咨询添加时请备注来意，并注明您的姓名-公司-职位药事纵横上下两册扫码添加秘书参与转发送活动注：最终解释权归主办方所有概况微球制剂的发展已历经30余载，自1985年美国首次报道微球制剂的研究以来，微球制剂便迅速成为全球药物研究的热点。2010年版的《中国药典》中，我国将微球制剂单独归为一种新剂型，作为新型制剂中的一个重要分支。微球制剂是指将药物分子巧妙地包埋或吸附在聚合物的表面，利用皮下注射或深部肌肉注射的方式进入人体，通过载体的快速释放、药物的扩散作用以及聚合物的溶蚀降解等机制，以实现药物的高效载荷、提高包封率，并能够实现对病变部位的精准靶向给药[1]。缓释微球制剂以其作用时间长、给药次数少、能有效减少不良反应等特点，已成为药物制剂的主要剂型之一。扫描下方二维码限时免费报名 PART.1 缓释微球制剂介绍 01 优势 Advantage 缓释微球制剂是目前国内外缓控释制剂创新研发的重要方向，正引领着医药创新的潮流。这种制剂能够实现药物的精准控制释放，从数周到数月不等，有效地规避了血药浓度的剧烈波动，即所谓的“峰谷现象”，能够降低因药物浓度异常波动带来的不良反应风险。缓释微球制剂通过维持血药浓度在一个平稳的治疗范围内，延长了药物的作用时间，显著提升了患者的用药依从性。相较于传统制剂，该类型制剂减少了给药频率，使患者能够享受到更为便捷和舒适的用药体验，对于需要长期服药的慢性疾病患者来说，无疑是一大福音。随着制剂技术的不断进步，缓释微球制剂有望在精准医疗领域扮演更加关键的角色，为患者带来更加个性化和高效的治疗方案。 02 给药途径 Drug delivery route 缓释微球制剂具有多种给药途径，包括注射、口服、肺部和黏膜给药等。每种给药途径都有其独特的优势，不同尺寸的微球在给药途径和作用方式上也存在显著差异。注射给药是缓释微球制剂的主要方式，涵盖静脉注射、皮下注射和肌肉注射等。当采用静脉注射时，药物可以直接进入血液，显著提高生物利用度，但需要严格控制注射颗粒的尺寸，以避免血管堵塞。皮下注射和肌肉注射同样常见，颗粒尺寸可达到数百微米，较大的颗粒在注射后能够在注射部位停留一段时间，包埋或吸附在微球中的药物则随着微球的降解逐渐释放。肺部给药是一种非侵入性的给药方式，通过肺泡上皮的吸收，药物可以迅速进入血液循环。微球可作为长效递送系统的吸入剂，但需确保微球的尺寸和结构较为合适，较大密度和尺寸的微粒难以到达肺泡，而较小的颗粒则容易被巨噬细胞吞噬，从而影响药物的疗效。缓释微球制剂的多样化给药途径为药物的有效传递提供了灵活性选择，但在实际应用中需综合考虑每种途径的优缺点以及颗粒特性。 03 制备材料 Material preparation 缓释材料是缓释微球制剂的重要组成部分，也是制剂应用的关键，需要选择合适的材料以确保药物释放的剂量和速度符合临床治疗需求，理想的缓释微球制剂能够使药物在组织或体液中保持有效的药物浓度，以实现治疗效果。在材料选择方面，缓释微球制剂主要采用有机材料和无机材料。有机材料主要包括天然或合成的高分子聚合物，如聚乳酸-羟基乙酸共聚物（PLGA）、聚己内酯（PCL）、聚乳酸（PLA）等。这些材料具有良好的生物相容性和生物可降解性，通过控制聚合物的分子量和组成比例，可以调节药物的释放速率。无机材料如碳纳米材料、介孔硅酸盐材料等，因其独特的物理化学性质，在药物传递系统中展现出特有的潜力。在选择缓释微球制剂的材料时，需要综合考虑药物的化学性质、预期的释放动力学、生物相容性、稳定性以及成本等因素。此外，材料的降解速率和机制也需与药物治疗效果相匹配，以确保在整个治疗周期内药物浓度保持在有效范围内。扫描下方二维码限时免费报名 PART.2 缓释微球的应用多肽药物缓释微球制剂艾塞那肽微球（Bydureon）是一种多肽类降糖药物，属于肠促胰素分泌剂，主要通过与胰高血糖素样肽1受体结合，促进胰岛素的释放，起到降糖作用。该药物由Alkermes和Amylin公司共同开发，并在2012年获得美国FDA的批准，用于2型糖尿病的治疗，代表了全球首个获批上市的胰高血糖素样肽-1（GLP-1）类似物的缓释微球制剂，具有显著的降糖效果和较小的副作用[2]。与传统的每日两次注射给药方式相比，艾塞那肽微球的创新之处在于将给药频率降低到每周一次，提高了患者的顺应性。临床试验数据表明，每周一次的艾塞那肽微球不仅在降糖效果上显著优于每日一次的艾塞那肽注射液，效果还可以与利拉鲁肽相媲美，通过皮下注射或深部肌肉注射进入人体后，能够通过载体的快速释放、药物的扩散作用以及聚合物的溶蚀降解等方式，实现药物的持续释放。缓释微球制剂能够较好地解决多肽类药物半衰期短、生物利用度低的问题，还能在注射部位持续释放药物，维持稳定的血药浓度。蛋白药物缓释微球制剂 1995年，基因泰克采用微球技术开发了长效的重组人生长激素微球Nutropin Depot，这是全球第一个大分子蛋白质微球制剂，用于治疗儿童生长激素缺乏症[3]。该制剂在临床上证明了长效生长激素的安全性和有效性，但在2004年因生产成本高、患者耐受性问题以及注射部位不良反应等原因已经退市。此外LG公司推出的微球LAGH制剂，采用透明质酸作为包材，将生长激素在注射前被掺入悬浮在中链甘油三酯中的透明质酸钠微球中而制成，可以实现每周一次给药。通过将蛋白药物包裹在微球中，不仅保护了蛋白药物免受外界环境的破坏，提高了稳定性，还实现了对药物释放速度和模式的精确控制，缓释微球制剂的开发和应用，为蛋白药物的递送提供了新的可能性，但同时也面临着生产成本、患者耐受性和制剂技术等方面的挑战。小分子药物缓释微球制剂利培酮微球是首个非典型抗精神病药的长效制剂，由PLGA为膜材制备，改善了传统利培酮的给药频率，从每日两次降低到每两周一次肌肉注射一次。不仅提高了患者的顺应性，还有助于维持稳定的血药浓度，减少因血药浓度波动带来的副作用，从而提高治疗效果和安全性。 PART.3 小结历经多年发展，微球制剂在药物递送领域引起了革命性的变化，成为了备受关注的新型递送工具。缓释微球通过聚合物包裹药物，实现精准释放，减少给药次数，提高患者依从性、便利性。同时缓释微球可通过皮下或肌肉注射，减少血药浓度波动，降低不良反应，为慢性病治疗带来了便利，也更安全可靠，为人类慢性疾病提供了新的治疗策略。扫描下方二维码限时免费报名参考文献： [1] 中华人民共和国药典[M]. 国家药典委员会.中国医药科技出版社.2010 [2]LITH,CHANDRASHEKARA,BEIGA,et al.Characterization of attributes and in vitro performance of exenatide-loaded PLGA long-acting release microspheres[J].Eur J Pharm Biopharm,2021,158:401-409. [3] TAKURA K, HIROSE T, CREA R, et al.E xpression in Escherichia coli of a chemicaIly synthesized gene for the hormone so matostatin[J].Science, 1977, 198 (4321) :1056-1063. [4] 李茜，张云淑，严保平，等. 注射用利培酮微球（Ⅱ）治疗急性期精神分裂症患者的疗效及安全性研究［J］. 中国全科医学，2023.DOI：10.12114. 论坛议程 *上下滑动查看议程 12月3日 14:00-14:40 新型药物递送系统技术在纳米制剂中的应用黄永焯中国科学院上海药物研究所研究员 14:40-15:20 各类药物溶出方法在制剂研发中的应用及案例分析段云剑深圳市华溶分析仪器有限公司技术总监 15:20-15:40 茶歇 15:40-16:20 药物制剂新技术及新辅料的产业化 16:20-17:00 微球制剂研发策略与挑战张建涛中科院宁波材料所研究员 12月4日 14:00-14:40 口腔膜剂的技术瓶颈及解决策略陈芳医药先进制造国家工程研究中心研究员 14:40-15:20 缓控释制剂开发重难点剖析及案例分享梁超峰主任药师、高级工程师、广东省制药工程专委会，广州市控缓释制剂研究中心，广州市药物制剂新型制药技术重点实验室 15:20-16:00 微纳递药系统的创新发展与应用转化李永勇同济大学医学院教授参会报名注册报名：大会限时免费（人数限制，先注册先得，本次报名为预登记报名，报名审核通过后为报名成功！），食宿自理。参会报名咨询：吴女士 13186968078（微信同号）崔女士 13018913819（微信同号）文女士 18868801402（微信同号）扫描下方二维码限时免费报名

2024-10-30

·GlobeNewswire-Health Care

Lumos Pharma Announces Two Abstracts Accepted for Oral Presentation at the 62ⁿᵈ Annual ESPE Meeting 2024

AUSTIN, Texas, Oct. 30, 2024 (GLOBE NEWSWIRE) -- Lumos Pharma (NASDAQ:LUMO), a clinical stage biopharmaceutical company focused on therapeutics for rare diseases, announced today that updated analyses of data from the Company’s Phase 2 OraGrowtH210 and OraGrowtH212 clinical trials have been accepted for oral presentation at the 62nd Annual European Society for Paediatric Endocrinology Meeting, or ESPE 2024, to be held November 16-18, 2024 in Liverpool, UK. Two abstracts on data from the OraGrowtH Trials evaluating oral LUM-201 for Pediatric Growth Hormone Deficiency (PGHD) will be presented on Sunday, November 17th, by Elżbieta Petriczko, MD, PhD, Pomeranian Medical University, Szczecin, Poland, and on Monday, November 18th, by Peter E. Clayton, MD, University of Manchester, Manchester, UK, respectively. LUM-201 Oral Abstract Presentations: Sunday, November 17th – Session: Free Communications 7: GH and IGFs – 10:50AM Local Time FC7.6 Abstract entitled, Growth, IGF-1 and IGFBP-3 Responses to Oral LUM-201 in OraGrowtH210 and OraGrowtH212 Trials in Pediatric Growth Hormone Deficiency (PGHD) Over 12 to 24 Months on Treatment (Elżbieta Petriczko, MD, PhD, et al) Monday, November 18th – Session: Free Communications 15: Late Breaking – 9:40AM Local Time FC15.2 Abstract entitled, Amount and Pattern of Pulsatile GH Secretion Induced by the Oral Growth Hormone Secretagogue LUM-201 Is Related to Growth and IGF-1 Responses in Moderate Pediatric Growth Hormone Deficiency (PGHD) (Peter E. Clayton, MD, et al) About Lumos Pharma Lumos Pharma, Inc. is a clinical stage biopharmaceutical company focused on the development and commercialization of therapeutics for rare diseases. The Company was founded and is led by a management team with longstanding experience in rare disease drug development. Lumos Pharma’s lead therapeutic candidate, LUM-201, is a novel, oral growth hormone (GH) secretagogue, seeking to transform the ~$4.7B global GH market from injectable to oral therapy. LUM-201 is currently being evaluated in multiple Phase 2 clinical studies in Pediatric Growth Hormone Deficiency (PGHD) and has received Orphan Drug Designation in both the US and EU. For more information, please visit https://lumos-pharma.com/. Investor & Media Contact: Lisa MillerLumos Pharma Investor Relations512-792-5454ir@lumos-pharma.com Source: Lumos Pharma, Inc.

临床2期孤儿药

2024-10-30

·GlobeNewswire-Health Care

A Race Against Blindness Grants $1 Million Supporting Axovia Therapeutics’ Planned Clinical Trial for Bardet-Biedl Syndrome (BBS)

PHOENIX, Oct. 30, 2024 (GLOBE NEWSWIRE) -- A Race Against Blindness, a nonprofit organization dedicated to funding sight-saving research for children, is pleased to announce a $1 million grant to Axovia Therapeutics to support the company’s lead program, AXV-101. This marks an important step toward developing a treatment to address blindness and obesity associated with Bardet-Biedl Syndrome 1 (BBS1), a rare genetic disorder with no current curative options. The funding from A Race Against Blindness will directly support Axovia's near-term milestones associated with the initiation of the clinical study and initial patient enrollment in the United Kingdom in mid-2025 to treat blindness caused by BBS1 mutations. Axovia Therapeutics’ future plans include initiation of a clinical trial in the United States and progression of the program to also treat obesity. BBS is a devastating genetic disorder associated with primary cilia dysfunction. One of the hallmarks is retinal degeneration commonly presenting as retinitis pigmentosa. The other hallmark is hyperphagia resulting in morbid obesity, among other complex clinical manifestations. Of the many genes known to be associated with BBS, pathogenic variants in the BBS1 gene are most commonly encountered. Axovia is developing a genetic therapy for those harboring two mutations in the BBS1 gene. There is no cure for BBS, and its prevalence ranges between one in 70,000 and one in 100,000 in Europe and North America, with up to 10 times this prevalence in the Middle East. “Our $1 million grant to Axovia is a significant step for the BBS community, and we are optimistic about Axovia’s groundbreaking clinical research. However, the path to treating RP/BBS is challenging and there is still much work to be done. A Race Against Blindness is committed to supporting the effort to raise the necessary funds to ensure the execution of the AXV-101 clinical trial and potentially bring a new therapeutic option to BBS patients like our son, Luke,” said Dr. Stephen Johnston, Co-Founder and President. “We are advancing the AXV-101 program and preparing to initiate the first clinical study in mid-2025. We are immensely grateful for this funding from A Race Against Blindness,” said Professor Phil Beales, Chief Executive Officer and Co-Founder at Axovia Therapeutics. “By utilizing gene therapy to address the loss of sight and obesity experienced by patients with BBS1, we believe our approach will have a profound and dramatic impact on the health and well-being of this community. We look forward to generating clinical data for this program.” With the goal of initiating clinical studies in mid-2025, A Race Against Blindness will continue aggressive efforts to fundraise, engage the scientific community, and raise global awareness of BBS. Media Contact:Stephen Johnston, M.D., Co-Founder and PresidentSteve@ARaceAgainstBlindness.org (520) 548 - 1807

临床研究