Laron综合征

定义 英文原文（暂无中文）

Laron syndrome is a congenital disorder characterized by marked short stature associated with normal or high serum growth hormone (GH) and low serum insulin-like growth factor-1 (IGF-I) levels which fail to rise after exogenous GH administration.

别名

生长激素抵抗所致矮小

基本事实

遗传方式

常染色体隐性

发病年龄

婴儿期、新生儿期

患病率

1-9 / 1 000 000（Europe）

相关基因 1

临床表型 29

近两年的全球研究 101L2

2024/08 起在 Europe PMC 检索所得，按发表时间倒序显示最近 20 篇。标题未译成中文——自动翻译需要接入 LLM 服务，尚未引入。

• 2026-07预印本
  The Laron Syndrome Mouse Model Reveals a Potential Contribution of Methylglyoxal-Derived Glycative Stress to IGF-1-Driven Prostate Cancer Progression
  · DOI
• 2026-07
  LARON SYNDROME. (Growth Hormone Insensitivity) Geographical distribution and relationship to the genetic defects in the growth hormone receptor gene
  The Journal of clinical endocrinology and metabolism · DOI · Europe PMC
• 2026-06
  Comparison Between Chronological and Bone Age at Menarche in Girls with Laron Syndrome
  Children (Basel, Switzerland) · DOI · Europe PMC
• 2026-06综述
  Real world data on rhIGF-1 therapy in children with severe primary IGF-I deficiency - The European experience
  Growth hormone & IGF research : official journal of the Growth Hormone · DOI · Europe PMC
• 2026-06
  Alexander von Humboldt and the little women of Loja
  Growth hormone & IGF research : official journal of the Growth Hormone · DOI · Europe PMC
• 2026-05开放获取
  Sex-specific roles of insulin-like growth factor-1, lean mass and fat mass in type 2 diabetes prevention: Mendelian randomization studies in Western and East Asian populations
  Cardiovascular diabetology. Endocrinology reports · DOI · Europe PMC
• 2026-05开放获取
  GH-resistant (Laron) mice: gene therapy with a liver-specific GH receptor causes unbalanced upregulation of female-biased and growth-related genes
  Frontiers in endocrinology · DOI · Europe PMC
• 2026-05系统综述综述开放获取
  Growth Hormone and Brain Regeneration: Evidence from Clinical Studies in Dementia, Traumatic Brain Injury, and Stroke: A Systematic Review
  International journal of molecular sciences · DOI · Europe PMC
• 2026-04开放获取
  Utilization of genetic biomarkers for childhood stunting surveillance and early detection in Southeast Asia: a systematic review
  Annals of pediatric endocrinology & metabolism · DOI · Europe PMC
• 2026-04
  Age and sexual maturation at Spermarche in boys with Laron syndrome
  Journal of pediatric endocrinology & metabolism : JPEM · DOI · Europe PMC
• 2026-04综述
  Growth hormone receptor blockade in cancer treatment
  Growth hormone & IGF research : official journal of the Growth Hormone · DOI · Europe PMC
• 2026-04
  Body proportion is not affected by sex in LARON syndrome
  Growth hormone & IGF research : official journal of the Growth Hormone · DOI · Europe PMC
• 2026-04开放获取
  MCT4 deficiency suppresses tumor incidence and metastasis by downregulating IGF1 expression and enhancing anti-tumor immunity
  Communications biology · DOI · Europe PMC
• 2026-04开放获取
  Zebrafish gon4la mutants recapitulate human GON4L-related growth disorders and reveal novel metabolic organs abnormalities
  Scientific reports · DOI · Europe PMC
• 2026-04开放获取
  Targeting Mitochondrial Stress Responses: Terbinafine and Miglustat as Novel Lifespan and Healthspan Modulators
  Aging cell · DOI · Europe PMC
• 2026-04荟萃分析开放获取
  Long-acting growth hormone for treating growth hormone deficiency in children: a meta-analysis of randomized controlled trials focusing on changes in body mass index
  The Journal of clinical endocrinology and metabolism · 被引 2 · DOI · Europe PMC
• 2026-03综述
  The IGF-1 senescence switch: a biphasic model for SASP-driven aging and precision senomodulation
  Cytokine · DOI · Europe PMC
• 2026-03
  A cartilage-targeted IGF-1-antibody fusion protein as a new therapeutic approach for IGF-1 deficiency
  Molecular therapy : the journal of the American Society of Gene Therap · DOI · Europe PMC
• 2026-03
  Low or Low-Normal Insulin-Like Growth Factor 1 After Traumatic Brain Injury: Interpretation and Implications
  Journal of neurotrauma · DOI · Europe PMC
• 2026-03综述开放获取
  Growth factor applications and clinical translation: advances and challenges
  Annals of medicine · DOI · Europe PMC

境外已获批用于本病的药物 1L2

欧盟 1 项、美国 0 项。同一药物在两地各批一次的，会分别列出。

• Increlex欧盟2007-08-02
  mecasermin
  官方记录

数据来自欧洲药品管理局（EMA）的药品与孤儿药资格公开导出表，以及美国 FDA 孤儿药资格数据库。两边口径不同：欧盟一侧取的是当前状态仍为「已授权」的药品；美国一侧记录的是「曾获批准」这一事实，FDA 的公开表不追踪药物此后是否退市（例如 Relyvrio 于 2024 年撤市，表中仍记为已获批）。请以官方记录页为准。

在中国开展的临床试验 1L2

按病名在 ClinicalTrials.gov 检索、并校验研究中心含中国大陆而来。登记状态不等于现在真的能入组——务必按 NCT 号到原站核实，并与主治医生商量。

当前没有检索到登记为可入组的试验。

外部标识与链接