The objective of this post-marketing surveillance (PMS) study is to assess the safety and effectiveness of CRYSVITA injection 10, 20, and 30mg, equivalent to in routine clinical settings

开始日期2023-08-11

申办/合作机构

Kyowa Kirin Korea Co., Ltd.

NCT05509595

/ Completed临床2期IIT

A Phase 2 Study of Burosumab for Fibroblast Growth Factor-23 Mediated Hypophosphatemia in Fibrous Dysplasia

Background:
Fibrous dysplasia (FD) is a disorder that affects bone growth. Affected bone tissue is weakened, and people with FD are prone to deformities, fractures, and other problems. People with FD may also have low blood phosphate levels. This can make bones even weaker. Better treatments are needed.
Objective:
To test a study drug (burosumab) in people with FD who have low blood phosphate levels.
Eligibility:
People aged 1 year or older who have FD and low blood phosphate levels.
Design:
Participants will visit the NIH 3 times in 48 weeks. Each visit will last 5 to 7 days.
Participants will self-inject burosumab under the skin in their belly, upper arm, or thigh. They (or a caregiver) will do this at home 1 or 2 times a month. They will be trained in person on how to inject the drug. Home injections will be guided via telehealth.
During NIH visits, participants will have a physical exam with blood and urine tests. They will have x-rays of different parts of their body. They will have a radioactive tracer injected into their vein; then they will have a bone scan. They will have tests to assess their strength, walking, and movement. They will complete questionnaires about their pain, mobility, and fatigue levels.
Adult participants may have bone biopsies. These will be done under anesthesia with sedation. Small samples of FD-affected bone will be removed for study.
Between NIH visits, participants will go to a local laboratory for blood and urine tests.
Child participants will have an additional follow-up visit 2 weeks after the final NIH visit.

开始日期2022-12-07

申办/合作机构

National Institute of Dental & Craniofacial Research

100 项与布罗索尤单抗相关的临床结果

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100 项与布罗索尤单抗相关的转化医学

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100 项与布罗索尤单抗相关的专利（医药）

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322

项与布罗索尤单抗相关的文献（医药）

2027-01-01·MOLECULAR AND CELLULAR ENDOCRINOLOGY

Fibroblast growth factor 23 (FGF23): From synthesis to cleavage

Review

作者: Wang, Qi ; Yuan, Shusen ; Meng, Chao ; Li, Baowei ; Ji, Hongjie

Fibroblast growth factor 23 (FGF23) maintains phosphate and vitamin D homeostasis. In the kidney, recognition of FGF23 is mediated by fibroblast growth factor receptors (FGFRs), α-Klotho, and heparan sulfate. Structural studies indicate that an FGF23-FGFR-α-Klotho recognition unit recruits a second FGFR with assistance from heparan sulfate to form an asymmetric 1:2:1:1 complex that activates Ras-mitogen-activated protein kinase (MAPK) signaling. Circulating intact FGF23 reflects transcription, post-translational processing, secretion, and clearance. In FGF23-producing cells, polypeptide N-acetylgalactosaminyltransferase 3 (GALNT3)-mediated O-glycosylation at Thr178 protects the cleavage site, whereas phosphorylation of Ser180 by FAM20C, a Golgi-associated secretory pathway kinase, limits this protection. Furin and related proprotein convertases cleave FGF23 within the secretory pathway, although their in vivo contributions remain unresolved. In cell-free experiments, tissue-type and urokinase-type plasminogen activators directly cleave recombinant FGF23; mouse data also implicate the plasminogen activator inhibitor-1 axis, but its quantitative contribution to the human circulating pool is unknown. Receptor availability further shapes signaling. Although osteocytes and osteoblasts are the principal endocrine sources, human skin cells express and secrete FGF23 in vitro. Their response to 1,25-dihydroxyvitamin D₃ depends partly on the vitamin D receptor, but whether skin contributes to circulating FGF23 remains undefined. Chronic kidney disease, autosomal dominant hypophosphatemic rickets, hyperphosphatemic familial tumoral calcinosis, X-linked hypophosphatemia, Raine syndrome, and ectonucleotide pyrophosphatase/phosphodiesterase 1 (ENPP1) deficiency illustrate the relationships among FGF23 processing, bone-matrix signaling, and pyrophosphate homeostasis. This review integrates these mechanisms and evaluates burosumab, selective FGFR inhibition, C-terminal peptides, small-molecule antagonists, and ENPP1 enzyme replacement according to current evidence.

2026-12-01·BONE

Three Generations of X-Linked Hypophosphataemia: The Inter-generational Impact of Burosumab Across the Lifespan

Article

作者: Girgis, Christian M ; Bilal, Maria ; Poon, Myra ; Kim, Albert ; Kumar, Shejil

X-linked hypophosphataemia (XLH) is a rare, lifelong heritable metabolic bone disorder characterized by fibroblast growth factor 23 (FGF23) excess, chronic hypophosphataemia, renal phosphate wasting, progressive skeletal deformities and impaired quality of life. Burosumab, targeted anti-FGF-23 therapy, is approved for use in XLH across the lifespan. Clinical trials have demonstrated benefit, although distinct outcomes in response to treatment vary depending on whether initiated during childhood, adolescence or adulthood. We uniquely describe three patients with XLH across three generations within a single family, each initiated on burosumab at different stages of life. This familial case study highlights the broad phenotypic spectrum of XLH, and differential efficacy profile of burosumab across various stages, such as pre- and post-growth plate closure or in the presence of established musculoskeletal morbidity. We also demonstrate the unique inter-generational impact of burosumab in XLH, as a targeted novel treatment available for patients with a dominantly inherited disorder across the lifespan.

2026-09-30·JBMR Plus

Burosumab as bridging therapy in autosomal dominant hypophosphatemic rickets with femoral fracture nonunion and iron deficiency

Article

作者: Rakusa, Matej ; Kocjan, Tomaz

Abstract:

Autosomal dominant hypophosphatemic rickets (ADHR) is a rare genetic disorder, characterized by fibroblast growth factor 23 (FGF23)-mediated renal phosphate wasting and impaired bone mineralization. Iron deficiency in ADHR increases FGF23 production and can worsen hypophosphatemia. Evidence to guide optimal management in severe adult cases remains limited. We report the use of burosumab as bridging therapy in a 19-yr-old woman with genetically confirmed ADHR (FGF23 p.Arg176Trp), severe osteomalacia, iron-deficiency anemia, and a low-energy femoral diaphyseal fracture complicated by nonunion despite conventional therapy. Treatment with burosumab resulted in rapid clinical improvement, with marked reduction in musculoskeletal pain and stiffness, and restoration of independent ambulation within four months. BMD increased substantially (+11.7% at the LS and +14.2% at the total hip at 5 mo; +63.8% and +55.5% from baseline at approximately 11 mo, respectively), while trabecular bone score remained within the normal range, a pattern consistent with improved mineralization and preserved trabecular texture. Serum phosphate increased but did not consistently normalize at trough, prompting dose escalation. At 8 mo, the burosumab dose was increased to 50 mg (1.25 mg/kg) due to persistent hypophosphatemia; with concurrent oral iron replacement, transient hyperphosphatemia (1.61 mmol/L) occurred and was managed by delaying the next dose by 14 d and reducing the dose back to 1 mg/kg. The patient also underwent additional surgical intervention, which may have contributed to the overall outcome; radiographic callus formation was evident 5 mo post-surgery. To our knowledge, this is the first full-length report describing burosumab use in genetically confirmed ADHR with longitudinal biochemical and densitometric follow-up, and it highlights the importance of careful monitoring of phosphate and iron indices when combining therapies.

148

项与布罗索尤单抗相关的新闻（医药）

2026-10-14

·GlobeNewswire-Health Care

Three-Year Real-World Study Compares Burosumab Treatment with Oral Phosphate and Active Vitamin D in Adults with XLH

Publication in the Journal of Bone and Mineral Research offers new insight into the long-term XLH treatment experience PRINCETON, N.J., Oct. 14, 2026 (GLOBE NEWSWIRE) -- Kyowa Kirin, Inc., a wholly owned subsidiary of Kyowa Kirin Co., Ltd. (TSE:4151, Kyowa Kirin), today announced the publication of three-year real-world findings from an interim analysis of the XLH Disease Monitoring Program (DMP) in the Journal of Bone and Mineral Research. The analysis assessed serum phosphate levels and patient-reported pain, stiffness, and physical function at years 1 and 3 among adults with X-linked hypophosphatemia (XLH) treated with burosumab or oral phosphate and active vitamin D (Pi/D). Read the full study here. “Real-world evidence in rare diseases like XLH is invaluable, helping to deepen our understanding of the patient experience and treatment outcomes beyond clinical trials,” said lead author Steven W. Ing, MD, MSCE, Professor of Clinical Medicine, Division of Endocrinology, Diabetes and Metabolism, The Ohio State University Wexner Medical Center. “These findings add to the broader evidence base for burosumab and can help inform clinical decision-making and the ongoing evolution of care for adults living with XLH.” XLH is a rare, lifelong, progressive genetic disease that causes excessive phosphorus loss, weakening the bones and muscles in children and adults, leading to skeletal deformities.1 The XLH DMP is a prospective, 10-year, observational study of adults and children with XLH in the US, Canada, and Latin America. It is designed to characterize XLH disease presentation and progression, and to prospectively assess changes over time in biochemical, clinical, and patient/caregiver-reported outcomes in a representative population. No randomized studies have directly compared treatment with burosumab, a monoclonal antibody targeting FGF23, with oral phosphate and active vitamin D in adults with XLH. This three-year analysis included 109 adults enrolled in the XLH DMP: 54 who received burosumab and 55 who received Pi/D. “For people living with a rare, lifelong condition such as XLH, it is essential to understand how treatments perform over time,” said Angela Williams, PhD, Vice President, Global Head of Health Economics and Outcomes Research at Kyowa Kirin. “By following patients longitudinally and incorporating patient-reported measures, the XLH Disease Monitoring Program provides additional context for the clinical data supporting burosumab use in adults with XLH.” In addition to the XLH Disease Monitoring Program, Kyowa Kirin is advancing real-world insight generation through the International XLH Registry in Europe and the SUNFLOWER study in Japan and Korea. About CRYSVITA® (burosumab-twza) Injection CRYSVITA is a recombinant fully human monoclonal IgG1 antibody, discovered by Kyowa Kirin, which binds to and inhibits the biological activity of FGF23, the underlying cause of hypophosphatemia in XLH. By blocking FGF23, CRYSVITA helps to restore phosphorus reabsorption in the kidneys and increase the production of active vitamin D, which enhances intestinal absorption of phosphate and calcium. U.S. CRYSVITA Indication CRYSVITA is a fibroblast growth factor (FGF23) blocking antibody indicated for the treatment of X-linked hypophosphatemia (XLH) in adult and pediatric patients 6 months of age and older. Important Safety Information CONTRAINDICATIONS CRYSVITA is contraindicated: In concomitant use with oral phosphate and/or active vitamin D analogs (e.g., calcitriol, paricalcitol, doxercalciferol, calcifediol) due to the risk of hyperphosphatemia. When serum phosphorus is within or above the normal range for age. In patients with severe renal impairment or end stage renal disease because these conditions are associated with abnormal mineral metabolism. WARNINGS AND PRECAUTIONS Hypersensitivity Hypersensitivity reactions (e.g., rash, urticaria) have been reported in patients with CRYSVITA. Discontinue CRYSVITA if serious hypersensitivity reactions occur and initiate appropriate medical treatment. Hyperphosphatemia and Risk of Nephrocalcinosis Increases in serum phosphorus to above the upper limit of normal may be associated with an increased risk of nephrocalcinosis. For patients already taking CRYSVITA, dose interruption and/or dose reduction may be required based on a patient’s serum phosphorus levels. Hypercalcemia Increases in serum calcium have been reported in patients treated with CRYSVITA. Patients with risk factors such as pre-existing hyperparathyroidism, prolonged immobilization, dehydration, hypervitaminosis D, or renal impairment, are at higher risk of hypercalcemia. Monitor these patients for serum calcium and parathyroid hormone levels before and during CRYSVITA treatment for moderate to severe hypercalcemia. In patients with moderate to severe hypercalcemia, CRYSVITA should not be administered until hypercalcemia is adequately managed. Injection Site Reactions Administration of CRYSVITA may result in local injection site reactions. Discontinue CRYSVITA if severe injection site reactions occur and administer appropriate medical treatment. ADVERSE REACTIONS Pediatric Patients Adverse reactions reported in 10% or more of CRYSVITA-treated pediatric XLH patients across three studies are: pyrexia (55%, 44%, and 62%), injection site reaction (52%, 67%, and 23%), cough (52%), vomiting (41%, 48%, and 46%), pain in extremity (38%, 46%, and 23%), headache (34% and 73%), tooth abscess (34%, 15%, and 23%), dental caries (31%), diarrhea (24%), vitamin D decreased (24%, 37%, and 15%), toothache (23% and 15%), constipation (17%), myalgia (17%), rash (14% and 27%), dizziness (15%), and nausea (10%). Adult Patients Adverse reactions reported in more than 5% of CRYSVITA-treated adult XLH patients and in at least 2 patients more than placebo in one study are: back pain (15%), headache (13%), tooth infection (13%), restless legs syndrome (12%), vitamin D decreased (12%), dizziness (10%), constipation (9%), muscle spasms (7%), and blood phosphorus increased (6%). Spinal stenosis is prevalent in adults with XLH, and spinal cord compression has been reported. It is unknown if CRYSVITA therapy exacerbates spinal stenosis or spinal cord compression. USE IN SPECIFIC POPULATIONS There are no available data on CRYSVITA use in pregnant women to inform a drug-associated risk of adverse developmental outcomes. Serum phosphorus levels should be monitored throughout pregnancy. Report pregnancies to the Kyowa Kirin, Inc. Adverse Event reporting line at 1-844-768-3544. There is no information regarding the presence of CRYSVITA in human milk or the effects of CRYSVITA on milk production or the breastfed infant. Therefore, the developmental and health benefits of breastfeeding should be considered along with the mother’s clinical need for CRYSVITA and any potential adverse effects on the breastfed infant from CRYSVITA or from the underlying maternal condition. PATIENT COUNSELING INFORMATION Advise patients not to use any oral phosphate and/or active vitamin D analog products. Instruct patients to contact their physician if hypersensitivity reactions, injection site reactions, and restless legs syndrome induction or worsening of symptoms occur. You may report side effects to the FDA at (800) FDA-1088 or www.fda.gov/medwatch. You may also report side effects to Kyowa Kirin, Inc. at 1-844-768-3544. For important risk and use information, please see the full Prescribing Information for CRYSVITA. About X-linked hypophosphatemia (XLH) X-linked hypophosphatemia is a rare, lifelong, genetic disease that can impact the bones and muscles in both children and adults. In individuals with XLH, the body doesn't hold on to enough phosphorus, which is an essential mineral for bone health. This is due to the production of excess fibroblast growth factor 23 (FGF23), causing the body to release too much phosphorus through the urine. When phosphorus levels are too low (hypophosphatemia), it can cause the softening and weakening of growing bones in children (rickets) and of mature bones in adults (osteomalacia).1 In children, XLH typically appears as bowed legs or knock knees. Over time, bone weakening can also lead to bone abnormalities in the legs, delayed growth, and short stature. In adults, XLH may cause osteomalacia, fractures and pseudo-fractures, and hypophosphatemia.1 About the XLH Disease Monitoring Program The XLH DMP is an international, prospective, 10-year, longitudinal, observational study (NCT03651505) of adults and children with XLH in the US, Canada, and Latin America designed to characterize XLH disease presentation and progression, and prospectively assess changes over time in biochemical, clinical, and patient/caregiver-reported outcomes in a representative population. The program steering committee includes clinical experts in XLH, patient advocates from the XLH Network, and representatives from Kyowa Kirin and Ultragenyx Pharmaceutical. About Kyowa Kirin Kyowa Kirin aims to discover novel medicines with life-changing value. As a Japan-based Global Specialty Pharmaceutical Company, we have invested in drug discovery and biotechnology innovation for more than 70 years and are currently working to engineer the next generation of antibodies and cell and gene therapies with the potential to help patients with high unmet medical needs, such as bone & mineral, intractable hematological diseases/hemato-oncology, and rare diseases. A shared commitment to our values, to sustainable growth, and to making people smile unites Kyowa Kirin across the globe. You can learn more about the business of Kyowa Kirin at: https://www.kyowakirin.com References: Dahir K, et al. X-linked hypophosphatemia: a new era in management. J Endocr Soc. 2020;4(12):bvaa151. COR-US-CRY-0082 Oct 2026 Contact: Susan Thiele Head of Therapeutic Communications, North America media.northamerica.bt@kyowakirin.com

临床结果临床研究上市批准

2026-10-12

·刚刚BME

刚刚！《Bone Res》十大前沿方向解读！

刚刚BME · 前沿速递 2026.10.02 JOURNAL SCANBone Research三年 · 287 篇 · 标题与通讯作者全量分析刚刚！Bone Research三年十大前沿 What 287 titles published in Bone Research from Oct 2023 to Sep 2026 reveal about where skeletal research is heading — ten frontiers, one watch-list, and who is behind them BR Bone Research · 期刊三年全景扫描 2023.10.01 – 2026.09.30 · PubMed 全部题录这一期我们只看一本刊——Bone Research。三年间它发表了多少文章、在写什么、哪里在加速、通讯作者来自哪些国家？我们把 PubMed 收录的全部题录一条条读完，并逐篇核对了通讯作者，给出答案。 PART Ⅰ · 分析报告 01三年 287 篇，原创为主 2023 年 10 月至 2026 年 9 月，Bone Research 共发表研究论文与综述 287 篇，其中原创研究论文 210 篇（73%），综述与专家共识 77 篇。我们逐条阅读标题，按研究问题归入 11 个类别，篇数最多的 10 类即为本文的「十大方向」，骨肿瘤（11 篇）列为观察名单。 02发文量正在快速放大 67→102 2024 → 2025 年年发文增长 52% 103 2026 年前 9 个月已超过 2025 全年 73% 原创研究论文占比210 / 287 篇 1 项临床试验题名（纤维结构不良 · II 期） 2025 年发文量比 2024 年增长约五成，2026 年仍在加速。版面扩张的主体是原创研究——Bone Research 始终以原创性论文为主，综述与共识只占约四分之一，主要用于系统梳理领域进展。对投稿者而言，机制扎实的原创研究依然是本刊最核心的稿件。图1. Bone Research 各年度发文数（深色为研究论文，浅色为综述/共识）。2023 年仅含 10–12 月，2026 年统计至 9 月底。 03十一类主题，一个「基本盘」骨重塑与骨质疏松以 57 篇居首，占 20%；骨关节炎（37）、骨发育与遗传病（34）、骨修复与材料（32）构成第二梯队。值得注意的是椎间盘/脊柱（23）与病理性骨化/肌腱（16），这两类是本刊颇具辨识度、篇数稳定的主题。图2. 各主题篇数（n = 287）。条形末端浅色段为其中的综述/共识篇数；灰色为观察名单「骨肿瘤」。每篇只归入一个主题。 04标题里的高频「关键词」跨越主题看关键词，排在最前面的是力学（Piezo、刚度、机械负荷，35 篇）和衰老（31 篇），其后是神经/内感受、破骨细胞、单细胞与空间组学。线粒体（18 篇）出现得比很多人预期的更频繁，几乎贯穿 OA、椎间盘、骨衰老与骨质疏松。图3. 题名关键词出现篇数（一篇可命中多个关键词）。圆点面积与篇数成正比，深蓝 ≥20 篇，青色 9–19 篇，灰色 <9 篇。 05谁在加速比较 2024 与 2025 两个完整年份，增长最猛的是骨—代谢—器官对话（2→10）和骨免疫与炎症（2→10），骨关节炎也翻了一倍（8→16）。2026 年前 9 个月，骨重塑（18）、发育（13）与修复材料（13）都已追平或超过 2025 全年。图4. 十大方向的年度篇数。圆点面积与篇数成正比，蓝色标出 2024→2025 年翻倍且增量 ≥5 篇的方向。2026 年非完整年份。 06通讯作者：国际化正在加速 52% 2026 年海外参与论文占比首次超过一半 9→18 通讯作者覆盖国家数2024 → 2026 年 29→54 海外参与论文篇数2024 全年 → 2026 前 9 月 20 三年通讯作者覆盖国家总数我们逐篇识别了 287 篇论文的全部通讯作者（含共同通讯），按通讯作者所在国家把每篇论文归为「中国」「海外」或「中外合作」三类，再逐年统计。年份论文中国合作海外海外占比国家2024 年 67 38 2 2743% 92025 年 102 71 2 2930% 112026 年（1–9 月） 103 49 35152%18 注：「中国」指通讯作者均来自中国；「海外」指通讯作者均来自中国以外；「合作」指中外合作，即共同通讯作者同时来自中国和海外；海外占比 =（海外 + 中外合作）/ 当年论文数。2023 年仅含 10–12 月（15 篇），未列入。国际化在持续加强：海外参与的论文从 2024 年的 29 篇、2025 年的 31 篇，增至 2026 年前 9 个月的 54 篇，还没到年底就已接近前两年之和；占比在 2026 年升至 52%，首次超过一半。通讯作者覆盖的国家也从 2024 年的 9 个、2025 年的 11 个，扩展到 2026 年的 18 个。2025 年海外占比一度回落到 30%，主要是因为当年中国稿件大幅增加（38 → 71 篇），海外稿件本身仍在增长。图5. 每年中外文章占比（按通讯作者国家）。深青为中国，浅蓝为中外合作，蓝色为海外；右侧为海外参与占比与当年通讯作者覆盖国家数。2026 年统计至 9 月底。三年合计，中国（175 篇，61.0%）与美国（67 篇，23.3%）是两大稿源；德国 7 篇，日本、法国各 6 篇，韩国、英国、丹麦、澳大利亚各 4 篇，西班牙、比利时各 3 篇，加拿大、荷兰、瑞典、意大利各 2 篇，沙特阿拉伯、奥地利、新加坡、巴西、瑞士各 1 篇。其中西班牙、意大利、荷兰、奥地利、加拿大、沙特阿拉伯、新加坡、巴西 8 个国家是 2026 年首次出现在通讯作者名单中。图6. 三年通讯作者所在国家的论文篇数（n = 287）。括号内为占 287 篇的比例；共同通讯来自不同国家时，每个国家各计 1 篇；香港计入中国。 PART Ⅱ · 十大前沿方向01骨重塑与骨质疏松新靶点 57 篇02骨关节炎：线粒体、衰老与关节退变 37 篇03骨骼发育、生长板与遗传性骨病 34 篇04骨修复再生与生物材料 32 篇05骨—代谢—器官对话 24 篇06椎间盘退变、腰痛与脊柱 23 篇07骨骼衰老与年轻化 19 篇08骨免疫与炎症性骨病 18 篇09神经—骨轴与骨内感受 16 篇10病理性骨化与肌腱/附着点 16 篇 01 FRONTIER 01 骨重塑与骨质疏松新靶点 Remodelling & osteoporosis targets 57 篇硬骨素 · RANKL · 雌激素近两年 33/57原创 44 篇体量最大的「基本盘」：从「抑制破骨」走向对硬骨素、RANKL 来源和激素轴的精细拆解。 ◆ 解析硬骨素 loop3–LRP4 结合界面；硬骨素/DKK1 双特异性抗体改善慢性肾病骨骼 ◆ 空间成骨谱系转录组提出 TGF-β 与硬骨素联合靶向治疗骨质疏松 ◆ 停用抗 RANKL 后骨量与骨质双降；骨表面细胞被定位为 RANKL 主要来源与 denosumab 反弹推手 ◆ 骨髓脂肪前体细胞是成年骨吸收的主要调控者；FSHβ、雌激素-唾液酸化等绝经新机制 BME 机会「停药反弹」「慢病继发骨松」是临床真实痛点，缓释递送与序贯治疗的材料方案仍有大片空白。 ▍本方向最新 3 篇研究论文（共 57 篇） 1. Angiogenin mediates cell-cell fusion as a mitochondrial RNA processing enzyme2026 · 研究论文 · DOI: 10.1038/s41413-026-00545-1 2. Apoptosis of Mdm2-deficient osteocytes enhances osteogenesis through TRPM8-enriched apoptotic vesicles2026 · 研究论文 · DOI: 10.1038/s41413-026-00570-0 3. Bispecific antibody against sclerostin and DKK1 improves bone health and reduces bone marrow adipose tissue accumulation in experimental chronic kidney disease2026 · 研究论文 · DOI: 10.1038/s41413-026-00556-y 02 FRONTIER 02 骨关节炎：线粒体、衰老与关节退变 Osteoarthritis & joint degeneration 37 篇线粒体 · 力学 · 股骨头坏死近两年 27/37原创 24 篇 OA 研究的关键词已经从「炎症」换成「线粒体」和「衰老」，并把软骨下骨与股骨头坏死一起纳入视野。 ◆ 线粒体成为 OA 主线：SIRT3–PINK1–PKM2、SPI1 线粒体未折叠蛋白反应、两篇线粒体靶向疗法综述 ◆ Piezo1、基质刚度与机械过载，经 HDAC3、PDZK1 等通路驱动软骨细胞衰老 ◆ 单细胞揭示骨髓病变区衰老免疫图谱；骨细胞 GSDMD 缺失反而加速 OA ◆ 股骨头坏死：SIRT3 线粒体自噬-铁死亡、M2 外泌体调控中性粒胞外诱捕网 BME 机会「线粒体移植/线粒体工程化」与 OA 分型精准治疗，是材料与细胞工程最直接的接口。 ▍本方向最新 3 篇研究论文（共 37 篇） 1. Inhibition of ERK5 activity ameliorates osteoarthritis by suppressing NLRP3 inflammasome-mediated chondrocyte pyroptosis2026 · 研究论文 · DOI: 10.1038/s41413-026-00573-x 2. Inhibition of cyclin-dependent kinase 8 modulates chondrocyte transition to hypertrophy and provides in vivo protection against spontaneous osteoarthritis2026 · 研究论文 · DOI: 10.1038/s41413-026-00551-3 3. Microenvironmental stiffness directs microtubule perturbation in chondrocyte mitosis via ILK-refilinB/Smad3 axis2026 · 研究论文 · DOI: 10.1038/s41413-025-00491-4 03 FRONTIER 03 骨骼发育、生长板与遗传性骨病 Development, growth plate & rare disease 34 篇生长板 · 颅缝 · 成骨不全近两年 25/34原创 33 篇 Bone Research 的「特色栏目」：生长板干细胞、颅缝与一长串罕见骨病模型。 ◆ 人生长板静息区含静息干细胞样亚群；ApoE 可标记静息区全部软骨细胞 ◆ 成骨不全四种机制模型：Schnurri-3 抑制、Sp7 突变骨细胞树突缺陷、高骨量型 OI 等 ◆ 颅缝完整性与扩张：Fgfr3–Wnt 串话、PERK/TFEB 线粒体动态、RUNX2 维持颅底软骨联合 ◆ 纤维结构不良的 burosumab II 期试验——全刊题名中唯一一项临床试验 BME 机会罕见骨病 iPSC/类器官模型 + 基因编辑矫正（如 RUNX1 GWAS 变异编辑），是发育生物学走向转化的捷径。 ▍本方向最新 3 篇研究论文（共 34 篇） 1. A phase 2 trial of burosumab for treatment of fibroblast growth factor-23-mediated hypophosphatemia in children and adults with fibrous dysplasia2026 · 研究论文 · DOI: 10.1038/s41413-026-00523-7 2. FOXI3 establishes the ectodermal niche in pharyngeal arches for cranial neural crest cells and their lineages2026 · 研究论文 · DOI: 10.1038/s41413-025-00499-w 3. Fgfr3-Wnt signaling crosstalk is involved in maintaining cranial suture integrity2026 · 研究论文 · DOI: 10.1038/s41413-026-00558-w 04 FRONTIER 04 骨修复再生与生物材料 Bone repair & biomaterials 32 篇骨折愈合 · 类器官 · 水凝胶近两年 25/32原创 21 篇材料设计开始围绕「血凝块」「免疫」「时间同步降解」做文章，原创工作集中在骨类器官与供体差异。 ◆ 骨痂类器官揭示不同供体与生物学性别的软骨-骨转化差异 ◆ 时间同步降解骨黏合剂、矿化 DNA 四面体水凝胶兼顾免疫调节与成骨 ◆ 血管化骨类器官作为股骨头坏死的仿生平台；ITGA11 亚群预测供体成骨能力 ◆ 综述覆盖电场响应水凝胶、血凝块调控与细胞/蛋白/基因/mRNA 生物制剂 BME 机会「供体差异可预测」与「类器官作为疗效筛选平台」两条线交汇，正是骨类器官转化落地的位置。 ▍本方向最新 3 篇研究论文（共 32 篇） 1. A tissue engineering approach to regenerate the cranial suture skeletal stem cell niche with a multicompartment biomaterial scaffold2026 · 研究论文 · DOI: 10.1038/s41413-026-00539-z 2. APEX1, a transcriptional hub for endochondral ossification and fracture repair2026 · 研究论文 · DOI: 10.1038/s41413-025-00486-1 3. An ITGA11 expressing subpopulation as predictor for the donor-specific osteogenic capacity of stromal cells2026 · 研究论文 · DOI: 10.1038/s41413-026-00536-2 05 FRONTIER 05 骨—代谢—器官对话 Bone–metabolism–organ crosstalk 24 篇肠道菌 · 肥胖 · 糖尿病近两年 20/24原创 14 篇 2024→2025 年从 2 篇跳到 10 篇：肠道菌、肥胖与骨-肌对话成为增长最快的新板块。 ◆ 肠道菌：双歧杆菌 A6 经丁酸同时改善骨与肌；菌群可预测原发甲旁亢骨密度；绝经与老年骨松菌群不同 ◆ 肥胖小鼠骨髓脂肪扩张，经 PD-L1 造成骨髓免疫抑制并促破骨 ◆ 损伤骨分泌骨因子促糖尿病创面愈合；HIF 激活同时改善能量代谢与骨 ◆ 空间转录组解析骨—骨骼肌串话；HR-pQCT 影像组学刻画 CKD 皮质骨 BME 机会骨源因子与菌群代谢物是「可工程化」的系统性调控器，适合做口服/靶向递送与合成生物学方案。 ▍本方向最新 3 篇研究论文（共 24 篇） 1. Bacterial specificity of the gut microbiome predicts bone density in primary hyperparathyroidism2026 · 研究论文 · DOI: 10.1038/s41413-026-00529-1 2. Decoding cellular communication networks and signaling pathways in bone, skeletal muscle, and bone-muscle crosstalk through spatial transcriptomics in a young male mouse2026 · 研究论文 · DOI: 10.1038/s41413-026-00520-w 3. Expansion of bone marrow adipocytes in obese mice leads to PD-L1-driven bone marrow immunosuppression and osteoclastogenesis2026 · 研究论文 · DOI: 10.1038/s41413-026-00509-5 06 FRONTIER 06 椎间盘退变、腰痛与脊柱 Disc degeneration, back pain & spine 23 篇衰老 · 力学 · 腰痛近两年 17/23原创 17 篇椎间盘是 Bone Research 的「招牌病种」，并明确把研究终点指向腰痛。 ◆ 衰老与表观：Sirt6、分子伴侣介导自噬、D+Q senolytic、表观重编程恢复「年轻」髓核 ◆ H3K27me3 单细胞多组学重塑图谱；Piezo1 介导力学铁内流致髓核铁死亡 ◆ 腰痛：PTH 减少终板异常感觉神经长入；多学科腰痛诊疗专家共识 ◆ Akkermansia EV 递送效应蛋白减缓退变；脊髓损伤瘢痕与脊髓淋巴系统新视角 BME 机会以「疼痛」为终点的椎间盘再生、终板神经调控材料、衰老细胞清除，是骨科与疼痛医学的交叉蓝海。 ▍本方向最新 3 篇研究论文（共 23 篇） 1. Akkermansia muciniphila attenuates intervertebral disc degeneration via extracellular vesicle-mediated delivery of the effector protein B2UKX52026 · 研究论文 · DOI: 10.1038/s41413-026-00541-5 2. Dasatinib and quercetin senolytic treatment delays early onset intervertebral disc degeneration in SM/J mice2026 · 研究论文 · DOI: 10.1038/s41413-026-00526-4 3. PTH induced osteoblast Slit3 to decrease aberrant sensory innervation in degenerated vertebral endplates to relieve low back pain in mice2026 · 研究论文 · DOI: 10.1038/s41413-025-00488-z 07 FRONTIER 07 骨骼衰老与年轻化 Skeletal ageing & rejuvenation 19 篇衰老细胞 · 骨膜 · 老年骨折近两年 12/19原创 14 篇衰老研究直指一个临床问题：为什么老年人骨折愈合差，以及能否「返老还童」。 ◆ 骨膜线粒体 DNA 结构驱动衰老相关的骨修复不良 ◆ 中和肝源 ApoE 促进老年骨折愈合；巨噬细胞 grancalcin 诱导骨骼干细胞衰老 ◆ TFEB 自噬「年轻化」BMSC 并延长中年小鼠寿命；高尔基囊泡补充延缓骨衰老 ◆ 反直觉结果：小鼠增龄性骨丢失与肠道菌群无关；树鼩被提出为新的骨衰老模型 BME 机会老年骨折是最确定的需求场景：衰老微环境可调的植入物与「年轻化」生物制剂值得系统布局。 ▍本方向最新 3 篇研究论文（共 19 篇） 1. A RANKL/CXCR4B cell population accumulates in bone marrow and causes age-related osteoporosis in mice2026 · 研究论文 · DOI: 10.1038/s41413-026-00525-5 2. Neutralizing hepatic apolipoprotein E enhances aged bone fracture healing2026 · 研究论文 · DOI: 10.1038/s41413-025-00489-y 3. Periosteal mitochondria DNA structures drive aging-associated poor skeletal repair2026 · 研究论文 · DOI: 10.1038/s41413-026-00524-6 08 FRONTIER 08 骨免疫与炎症性骨病 Osteoimmunology & inflammatory bone disease 18 篇 RA · 骨髓炎 · 牙周炎近两年 15/18原创 14 篇免疫细胞代谢物、凋亡小体等「非经典信号」正在重写炎症性骨破坏的机制。 ◆ 炎性巨噬细胞来源衣康酸抑制 TET2，防止 RA 中破骨过度激活 ◆ 中性粒凋亡小体作为诱饵信号，在感染性骨髓炎中保护骨完整性 ◆ NF-κB 驱动成骨细胞转分化为炎性骨细胞样细胞；肠菌代谢物经 HDAC3 调控滑膜成纤维细胞 ◆ 强直性脊柱炎破骨前体 RPS17、牙周炎 RORα、多发伤与肺损伤诱发的全身骨丢失 BME 机会「代谢物/囊泡作为免疫调控药物」可直接转化为局部递送体系，感染性骨缺损是首选场景。 ▍本方向最新 3 篇研究论文（共 18 篇） 1. Inflammation driven by NF-ĸB compromises bone formation by inducing the differentiation of osteoblasts into atypical inflammatory osteocyte-like cells2026 · 研究论文 · DOI: 10.1038/s41413-026-00546-0 2. Neutrophil-derived apoptotic bodies function as decoy signals to preserve bone integrity during infectious osteomyelitis2026 · 研究论文 · DOI: 10.1038/s41413-026-00571-z 3. Sulfated chitosan mitigates acute lung injury induced bone loss via immunoregulation2026 · 研究论文 · DOI: 10.1038/s41413-025-00475-4 09 FRONTIER 09 神经—骨轴与骨内感受 Neuro-skeletal axis & interoception 16 篇骨内感受 · 感觉神经 · 中枢近两年 11/16原创 7 篇「骨骼内感受（skeletal interoception）」是本刊辨识度最高的概念：大脑在「感知」骨骼。 ◆ PGE2 骨骼内感受调控承重骨，并延伸到关节稳态、OA 疼痛、生物材料与类器官 ◆ 感觉神经分泌 Cyp40 直接促破骨；航天与失重模型中感觉神经调控骨稳态 ◆ 中性粒细胞启动伤害感受神经长入，促炎症消退与骨再生 ◆ 中枢层面：下丘脑交感通路与股骨头坏死；绿光经 GnIH 调控骨量 BME 机会「神经化」骨修复材料与内感受导向的镇痛-再生一体化疗法，是 BME 可以抢先定义的方向。 ▍本方向最新 3 篇研究论文（共 16 篇） 1. Neutrophil-initiated nociceptive ingrowth orchestrates inflammation resolution to potentiate bone regeneration2026 · 研究论文 · DOI: 10.1038/s41413-025-00481-6 2. Sensory nerves regulate bone homeostasis in response to mechanical unloading or reloading: evidence from a spaceflight experiment and ground-based mechanical unloading models2026 · 研究论文 · DOI: 10.1038/s41413-026-00563-z 3. GnIH secreted by green light exposure, regulates bone mass through the activation of Gpr1472025 · 研究论文 · DOI: 10.1038/s41413-024-00389-7 10 FRONTIER 10 病理性骨化与肌腱/附着点 Heterotopic ossification & tendon 16 篇异位骨化 · OPLL · 肌腱近两年 11/16原创 14 篇「不该长骨的地方长了骨」：异位骨化与后纵韧带骨化成为巨噬细胞与神经研究的新舞台。 ◆ 神经源性异位骨化中的脑源 EV；靶向外周神经预防创伤性异位骨化 ◆ 巨噬细胞 PRMT6–CCL2、坏死性巨噬细胞 Pak4 推动 HO；Clec3b⁺ 骨外细胞参与骨折与 HO ◆ OPLL：索拉非尼阻断 LOXL2 介导的血管化；巨噬细胞驱动机制综述 ◆ 肌腱：p16⁺ 细胞表观重编程促再生、代谢分型肌腱病、肩袖瘢痕与附着点祖细胞图谱 BME 机会附着点（腱-骨界面）再生与 HO 预防是同一枚硬币的两面，梯度材料大有可为。 ▍本方向最新 3 篇研究论文（共 16 篇） 1. Clec3bextraskeletal cells regulate fracture healing and heterotopic ossification2026 · 研究论文 · DOI: 10.1038/s41413-026-00532-6 2. Identification of a new population of Tnnprogenitors to form tendon enthesis fibrocartilage2026 · 研究论文 · DOI: 10.1038/s41413-026-00519-3 3. JMJD3-driven epigenetic reprogramming of p16-positive cells promotes tendon regeneration2026 · 研究论文 · DOI: 10.1038/s41413-026-00537-1 观察名单 · 骨肿瘤与骨转移（11 篇）篇数不多但质量集中：骨内膜生态位调控乳腺癌细胞休眠、黑色素瘤骨转移诱导骨细胞铁死亡、骨肉瘤空间多重免疫荧光预测生存、KRAS 突变肺癌溶骨转移，以及骨髓纤维化中脾源 ICAM2 导致骨硬化。 ▍本方向最新 3 篇研究论文（共 11 篇） 1. ADAR2 induces the differentiation of osteosarcoma cells by editing activity on IGFBP7: new implications for therapy2026 · 研究论文 · DOI: 10.1038/s41413-026-00516-6 2. The endosteal niche regulates breast cancer cell dormancy in bone: identification of new molecular determinants2026 · 研究论文 · DOI: 10.1038/s41413-026-00535-3 3. Spleen-derived circulating intercellular adhesion molecule 2 promotes osteosclerosis in chronic myelofibrosis2026 · 研究论文 · DOI: 10.1038/s41413-026-00575-9 PART Ⅲ · 编辑解读 ★编辑深度解读解读 1 「病种覆盖」是本刊最大的护城河椎间盘退变、后纵韧带骨化、股骨头坏死、异位骨化、肌腱病、颅缝与生长板疾病——这些在综合性顶刊中很少成为主角的骨科病种，在 Bone Research 里有稳定的专题密度。做这些病种的团队，本刊是天然的首选平台。解读 2 「衰老 × 线粒体 × 力学」是贯穿全刊的三条暗线三组关键词横跨 OA、椎间盘、骨质疏松、骨衰老与修复材料等多个方向。一篇稿件若能把其中两条线连起来（例如力学负荷如何经线粒体驱动细胞衰老），在机制层面最容易被本刊编辑识别为「主流问题」。解读 3 骨内感受是可以「抢定义」的新概念从承重骨、关节疼痛到生物材料与类器官，「骨骼内感受」在三年里被反复延展，并由多篇综述持续加固。对生物材料研究者而言，把材料设计的目标从「成骨」扩展为「被神经系统正确感知」，是一个尚未拥挤的切入角度。解读 4 转化证据仍是短板，也是机会 287 篇中题名明确为临床试验的只有 1 篇，类器官 4 篇、AI 3 篇。人源模型（类器官、器官芯片、患者来源样本）与临床数据驱动的研究，在本刊几乎没有竞争，是值得提前布局的方向。解读 5 国际化是本刊最清晰的上升曲线 2026 年前 9 个月，海外参与的论文已超过一半，通讯作者覆盖国家从 9 个翻倍到 18 个，仅今年就新增了来自欧洲、北美、中东、东南亚和南美的 8 个国家。中国学者依然是本刊最稳定的稿源，而越来越多的国际同行把 Bone Research 作为发表骨研究原创成果的平台——对国内团队而言，这里既是熟悉的主场，也是与国际同行同台的窗口。方法局限这份扫描的边界 ① 仅分析标题，未读摘要与全文；② 文章类型以 PubMed 标注为准（Review 与 Consensus Statement 计为综述/共识，其余计为原创研究）；③ 主题为编辑人工归类，部分论文可跨主题；关键词统计基于题名正则匹配，同一篇可计入多个关键词；④ 2023 年仅含 10–12 月、2026 年统计至 9 月底；⑤ 未评估单篇论文的引用或影响力；⑥ 通讯作者按 PubMed 单位字段中附有邮箱的作者识别（共同通讯全部计入），国家取每位通讯作者第一单位所在国，香港计入中国；一篇论文在同一国家只计 1 次，因此各国篇数之和略大于 287。刚刚BME 刚刚BME · 生物医学工程前沿速递数据来源：PubMed 收录的 Bone Research 题录（2023.10 – 2026.09）；通讯作者国家取自 PubMed 作者单位字段，各方向代表论文 DOI 见文中列表© 刚刚BME · 转载请注明来源 #BoneResearch#骨质疏松#骨关节炎#椎间盘退变#骨内感受#前沿趋势#通讯作者分析

临床1期

2026-10-12

·广州市妇女儿童医疗中心

广州妇儿中心亮相中华医学会第四届罕见病学术年会，多领域发声展实力

10月8日至11日，由中华医学会、中华医学会罕见病分会主办的中华医学会第四届罕见病学术年会暨2026中华医学会罕见病分会中青年学术会议在江苏南京召开。大会以罕见病领域新知识、新技术、新方法为核心，邀请全国罕见病领域知名专家进行专题报告、指南解读、病例讨论及论文交流，是国内罕见病领域规模最大、学术水平最高的年度盛会之一。国家儿童医学中心、广州市妇女儿童医疗中心（以下简称广州妇儿中心）作为全国罕见病诊疗协作网广东省牵头单位，组织多位专家参会，充分展现了广州妇儿中心在罕见病诊疗与研究领域的实力与风采。广州妇儿中心院长周文浩教授作为国内罕见病领域的资深专家，在“罕见病学科建设分会场”以《新生儿罕见病基因筛查与诊断》为题，深入探讨了基因组医学时代新生儿罕见病筛查的技术路径、临床挑战与未来方向，为儿童罕见病防治体系建设贡献“妇儿方案”。广州妇儿中心遗传与内分泌科刘丽教授作为中华医学会罕见病分会副主任委员，受邀担任主旨报告环节主持人，与北京大学第一医院袁云教授共同主持“干细胞的药代/药效及成药性研究”等重要大会报告。在中青年学术舞台上，张锐忠教授担任“罕见病中青年分会场”主席，并作《髓系细胞介导的胆道闭锁致病机制解析及诊疗新策略研究》专题发言，从基础研究到临床转化全方位展示了团队在胆道闭锁这一罕见肝病领域的最新成果。李俊教授在中青年分会场作《AI赋能罕见病诊断和致病变异发现》的专题报告，介绍人工智能技术在罕见病辅助诊断和基因变异解读中的创新应用，展现了广州妇儿中心在智慧医疗与罕见病交叉领域的前沿探索。程旸教授则担任罕见病学科建设分会场论文发言环节的主持人，以扎实的学术功底把控讨论节奏。在内分泌遗传代谢与质谱诊断领域，张文教授在“质谱技术助力罕见病筛查与精准诊疗”分会场作《质谱技术在儿童疑难罕见病诊疗中的临床应用实践》专题发言；唐诚芳副主任技师分享《新生儿串联质谱筛查在遗传代谢病死因早期发现中的应用价值研究》；吴鹏飞博士报告《儿童甲状旁腺功能减退症43例的分子病因分析》和《美多芭治疗TH基因变异所致酪氨酸羟化酶缺乏症的8年临床经验》；黄梓恩医生报告《蛋白敏感性高胰岛素血症性低血糖的临床特征、遗传谱及诊疗分析》；曾春华教授分享《X连锁低磷血症患儿生长模式的差异：常规治疗25年真实世界数据与Burosumab治疗3年结局的对比》和《铜蓝蛋白检测在婴幼儿肝豆状核变性筛查和诊断中的价值探讨》，展示了长期随访的珍贵数据。在溶酶体贮积病领域，孟岩教授在“溶酶体贮积病”分会场作《晚发型法布雷病综合治疗》专题发言，并担任专题会主持人；张敏娜护士报告《糖原累积病Ⅰ型患者营养管理的最佳证据总结》；宫薇薇博士带来《慢病毒介导的自体干细胞基因修饰治疗异染性脑白质营养不：单中心临床实践经验分享》这一前沿基因治疗成果。在外科领域，李军亮教授在“儿童卒中与罕见病”分会场作《婴幼儿硬脑膜动静脉瘘的特点和诊疗策略》专题发言；李文雷教授报告《单侧肺动脉缺如的外科治疗结果分析》。在护理领域，林艳主任在“罕见病护理分会场”作《非典型溶血尿毒综合征（aHUS）儿童靶向治疗全程规范化护理实践》专题发言；彭雯琦分别在血友病分会场和护理分会场报告了儿童血友病家庭治疗管理的相关研究；PICU罗毅护士长分享抗NMDA受体脑炎合并重症多器官损伤患儿的高级实践护理经验；血液肿瘤科黄海英护士长报告血友病患儿PICC置管的多学科全程管理策略。本次年会上，广州妇儿中心专家团队活跃在罕见病学科建设、基因筛查与诊断、内分泌遗传代谢、溶酶体贮积病、神经外科、护理、AI辅助诊断等多个领域，全面展示了医院在罕见病诊疗、研究和管理方面的丰硕成果。作为全国罕见病诊疗协作网广东省牵头单位和广东省儿童罕见病重点实验室依托单位，广州妇儿中心将继续秉承“以患者为中心”的理念，持续推进罕见病多学科诊疗模式，加强基因诊断与精准治疗能力建设，为罕见病患者提供更加优质、全周期的医疗服务，为推动中国罕见病事业发展贡献力量。罕见病医学中心作为国家儿童医学中心、全国罕见病专科排名第8、广东省罕见病诊疗协作网牵头单位，广州妇儿中心始终致力于为罕见病患儿家庭点燃希望之光。 ►国家平台，华南领航国家临床重点专科——小儿遗传病科广东省小儿遗传病重点实验室广东省罕见病医学中心广州市新生儿筛查中心国家卫生健康委罕见病协作网广东省牵头单位广东省医学会罕见病学分会主任委员单位广东省罕见病诊疗质量控制中心 ►专家团队 ►临床能力，数据说话 ►多学科协作（MDT）模式医院自2019年起设立罕见病专病门诊，每天接诊20-30例罕见病患儿，涵盖神经、遗传代谢、免疫、血液、肾脏、呼吸、内分泌等系统。典型案例 · Ommen综合征（极罕见重症联合免疫缺陷亚型）通过免疫、呼吸、感染、血液肿瘤科联合MDT，48小时内完成基因确诊并制定移植方案。 · 异染性脑白质营养不良成功实施华南地区首例自体造血干细胞基因治疗。 · 幼年型皮肌炎 264天个体化治疗，患儿顺利康复出院。 ►科研平台，转化引领 · 研究型病房：开展基因治疗、细胞治疗、酶替代治疗等前沿技术临床研究。 · 多组学融合诊断平台：整合基因组、代谢组、蛋白组、表观组等多维数据，实现精准分型。 · 产学研一体化平台：依托全球最大妇幼出生队列、百万级生物样本库，推动从机制发现到新药研发的全流程转化。 ►全生命周期管理 ►国际合作，共建共享 · 与美国波士顿儿童医院、英国伯明翰大学、费城儿童医院等15家国际顶尖机构建立长期合作。 · 成为国家卫健委“一带一路”妇幼健康示范培训基地。 ►就诊指引罕见病门诊地址：广州市妇女儿童医疗中心（珠江新城/增城医院）点击下方预约挂号：罕见病MDT门诊（上下滑动查看更多）文字丨遗传与内分泌科编辑丨陈文责编丨周密审核发布丨刘黔芳

100 项与布罗索尤单抗相关的药物交易

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外链

KEGG	Wiki	ATC	Drug Bank
D10913	布罗索尤单抗	M05BX05	DB14012

研发状态

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10 条最早获批的记录,

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适应症	国家/地区	公司	日期
FGF23阳性低磷血症	韩国	Kyowa Kirin Korea Co., Ltd.	2020-09-17
肿瘤源性骨软化症	美国	Kyowa Kirin, Inc. /Sancuso/	2020-06-18
低磷血症佝偻病	日本	Kyowa Kirin Co., Ltd.	2019-09-20
骨软化	加拿大	Kyowa Kirin Co., Ltd.	2019-01-28
家族性低磷酸盐血症性佝偻病	欧盟	Kyowa Kirin Holdings BV	2018-02-19
家族性低磷酸盐血症性佝偻病	冰岛	Kyowa Kirin Holdings BV	2018-02-19
家族性低磷酸盐血症性佝偻病	列支敦士登	Kyowa Kirin Holdings BV	2018-02-19
家族性低磷酸盐血症性佝偻病	挪威	Kyowa Kirin Holdings BV	2018-02-19

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适应症	最高研发状态	国家/地区	公司	日期
慢性疼痛	临床3期	美国	Ultragenyx Pharmaceutical, Inc.	2018-01-31
Jadassohn皮脂腺痣	临床3期	美国	Ultragenyx Pharmaceutical, Inc.	2018-01-31
低磷血症	临床1期	美国	Ultragenyx Pharmaceutical, Inc.	2019-07-01

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研究	分期	人群特征	评价人数	分组	结果	评价	发布日期


NCT04188964 (Pubmed \| Lancet Diabetes Endocrinol)	临床1/2期	家族性低磷酸盐血症性佝偻病	16	Burosumab	膚糧觸鑰夢構醖憲蓋衊(鑰醖鹹簾餘齋餘顧膚繭) = All participants had TEAEs with no differences in incidence across cohorts 齋願獵憲廠壓糧鬱顧簾 (範淵繭網衊憲餘膚遞糧 )	积极	2026-06-01
WCO_IOF_ESCEO2026	N/A	家族性低磷酸盐血症性佝偻病	6	Burosumab	鑰鏇襯膚淵鹹鏇鏇糧餘(顧艱鬱鏇艱願簾鏇選壓) = 糧鹹餘齋夢鑰鑰鑰廠鑰餘網齋糧觸觸遞艱窪齋 (糧構構鬱顧遞願遞齋醖 ) 更多	积极	2026-04-16
WCO_IOF_ESCEO2026	N/A	家族性低磷酸盐血症性佝偻病	6	Burosumab 1 mg/kg	鏇網願膚憲壓獵窪憲觸(鹽獵衊膚鏇觸襯窪餘鹹) = 網鹽廠膚壓齋餘餘憲鏇齋獵餘築膚鹽糧鏇獵壓 (網顧壓鑰憲鏇夢築蓋廠 ) 更多	积极	2026-04-16
NCT05509595 (CTgov)	临床2期	骨纤维发育不良 \| 低磷血症	12	Burosumab	網簾膚築淵膚鏇廠糧築 = 鑰網願鬱遞餘鬱餘壓鹹築網壓鑰壓糧鬱膚鏇顧 (積觸鏇鑰範鹽蓋鏇糧廠, 積廠積齋網鏇鏇網鹽選 ~ 積選齋積鬱顧網獵繭遞) 更多	-	2026-02-02
ESPE2025	N/A	家族性低磷酸盐血症性佝偻病	6	Burosumab	齋鹽積範鹹蓋鹹衊願築(襯糧鏇廠網鬱獵積築鏇) = 窪鹽鬱觸簾遞範獵構醖膚範觸膚憲醖夢襯範廠 (齋夢遞觸選壓壓製願顧 ) 更多	积极	2025-05-10
WCO_IOF_ESCEO2025	N/A	家族性低磷酸盐血症性佝偻病	22	phosphate salts and calcitriol	餘餘簾遞襯夢夢鬱遞壓(範願鬱製壓獵憲憲鏇壓) = 鏇鏇製觸積獵選襯鏇襯鏇糧衊鹽艱鹹淵遞齋蓋 (窪襯淵繭齋構夢構簾鏇, 2 ~ 5) 更多	积极	2025-04-10
ESPE2024	N/A	家族性低磷酸盐血症性佝偻病	19	Burosumab (X-linked hypophosphatemic rickets)	夢糧選築網鏇鹽襯製鏇(齋鏇範獵窪憲憲夢獵鹹) = 醖艱獵願製積淵顧襯醖簾鏇鹹願構鏇構窪繭膚 (鏇淵糧餘廠窪襯顧觸餘 ) 更多	积极	2024-11-16
ESPE2024	N/A	家族性低磷酸盐血症性佝偻病	24	Burosumab	壓構鏇鏇製簾餘觸繭簾(餘繭觸艱夢願鬱艱糧膚) = 鹹構糧積醖艱蓋襯積廠範繭餘窪糧壓網蓋襯積 (廠築糧膚願築鹹窪選窪, 0.2) 更多	积极	2024-11-16
ENDO2024	临床3期	家族性低磷酸盐血症性佝偻病 PHEX mutation	-	Burosumab	糧獵窪繭憲積膚鬱餘鬱(遞襯齋築簾築簾壓鹽鹹) = 鏇積蓋鑰廠憲築齋衊願鑰簾蓋構獵簾繭鬱觸齋 (餘積夢壓鏇淵鹹鏇獵糧 ) 更多	-	2024-06-01
NCT04146935 (CTgov)	临床4期	家族性低磷酸盐血症性佝偻病	10	Burosumab Injection [Crysvita]	鹽窪壓獵簾膚觸簾簾構(網廠鹹顧鹹範艱窪艱構) = 積窪鬱壓醖齋衊艱獵積廠窪鏇衊鹹繭襯艱獵獵 (蓋艱窪糧獵醖糧顧壓廠, 積築蓋衊襯夢繭襯鹹憲 ~ 醖襯鏇醖鬱憲鏇繭壓鏇) 更多	-	2023-08-24