ArticleExpert opinion on orphan drugs2015
New therapeutic targets in rare genetic skeletal diseases.
Article in Expert opinion on orphan drugs, 2015. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 21 papers.
What it found
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The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.
Who cites it
21 citing papers in PubMed.
- Differentiation of Pediatric Osteochondroma From Trevor's Disease and Successful Surgical Management: A Case Report.Case reports in orthopedics · 2026Article
- Modeling vascular dynamics at the initial stage of endochondral ossification on a microfluidic chip using a human embryonic-stem-cell-derived organoid.Regenerative therapy · 2025Article
- EndoCompass Project: Research Roadmap for Calcium and Bone Endocrinology.Hormone research in paediatrics · 2025Review
- Unraveling the complexity of skeletal dysplasias in the national health system.Frontiers in endocrinology · 2025Article
- Interventions for improving clinical outcomes and health-related quality-of-life for people living with skeletal dysplasias: an evidence gap map.Quality of life research : an international journal of quality of life aspects of treatment, care and rehabilitation · 2023Review
- Identification of variants in ACAN and PAPSS2 leading to spondyloepi(meta)physeal dysplasias in four Chinese families.Molecular genetics & genomic medicine · 2022Article
- Chondrodysplasias With Multiple Dislocations Caused by Defects in Glycosaminoglycan Synthesis.Frontiers in genetics · 2021Review
- Genetic Disorders of the Extracellular Matrix.Anatomical record (Hoboken, N.J. : 2007) · 2020Review
- Skeletal Dysplasias Caused by Sulfation Defects.International journal of molecular sciences · 2020Review
- New perspectives on the treatment of skeletal dysplasia.Therapeutic advances in endocrinology and metabolism · 2020Review
- New developments in chondrocyte ER stress and related diseases.F1000Research · 2020Review
- High Fidelity of Mouse Models Mimicking Human Genetic Skeletal Disorders.Frontiers in endocrinology · 2019Review
- Site-1 protease deficiency causes human skeletal dysplasia due to defective inter-organelle protein trafficking.JCI insight · 2018Article
- Epiphyseal hyperplasia caused by an ectopic ossification center of the distal tibia: presentation of a pediatric case with 13 years of radiological surveillance.Skeletal radiology · 2018Article
- 4-PBA ameliorates cellular homeostasis in fibroblasts from osteogenesis imperfecta patients by enhancing autophagy and stimulating protein secretion.Biochimica et biophysica acta. Molecular basis of disease · 2018Article
- The chaperone activity of 4PBA ameliorates the skeletal phenotype of Chihuahua, a zebrafish model for dominant osteogenesis imperfecta.Human molecular genetics · 2017Article
- Article
- Skeletal Dysplasias: Growing Therapy for Growing Bones.Frontiers in pharmacology · 2017Review
- Endoplasmic reticulum stress in chondrodysplasias caused by mutations in collagen types II and X.Cell stress & chaperones · 2016Review
- Cortical-Bone Fragility--Insights from sFRP4 Deficiency in Pyle's Disease.The New England journal of medicine · 2016Article
Corrections and comments
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Authors and funding
4 authors.
Funding
No grant is acknowledged in the PubMed record.
Abstract
PubMed holds no abstract for this paper.
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Identifiers
What OpenQuestion holds
Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.