ArticleExpert opinion on orphan drugs2013
From mysteries to medicines: drug development for fibrodysplasia ossificans progressive.
Article in Expert opinion on orphan drugs, 2013. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 23 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.
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Who cites it
23 citing papers in PubMed, 37 citations in OpenAlex.
- BCX9250 is a potent small-molecule inhibitor of Activin A Receptor Type 1 (ACVR1/ALK2) for fibrodysplasia ossificans progressiva.JBMR plus · 2026Article
- Article
- Pay Attention to the Osteochondromas in Fibrodysplasia Ossificans Progressiva.Orthopaedic surgery · 2024Article
- Review
- Functional comparison of human ACVR1 and zebrafish Acvr1l FOP-associated variants in embryonic zebrafish.Developmental dynamics : an official publication of the American Association of Anatomists · 2023Article
- Pathophysiology and Emerging Molecular Therapeutic Targets in Heterotopic Ossification.International journal of molecular sciences · 2022Review
- Pain Phenotypes in Rare Musculoskeletal and Neuromuscular Diseases.Neuroscience and biobehavioral reviews · 2021Review
- Targeting heterotopic ossification by inhibiting activin receptor‑like kinase 2 function (Review).Molecular medicine reports · 2019Review
- The role of Activin A in fibrodysplasia ossificans progressiva: a prominent mediator.Bioscience reports · 2019Review
- Difficult diagnosis and genetic analysis of fibrodysplasia ossificans progressiva: a case report.BMC medical genetics · 2018Article
- Fibrodysplasia ossificans progressiva: Basic understanding and experimental models.Intractable & rare diseases research · 2017Review
- TGF-β Family Signaling in Connective Tissue and Skeletal Diseases.Cold Spring Harbor perspectives in biology · 2017Review
- The traumatic bone: trauma-induced heterotopic ossification.Translational research : the journal of laboratory and clinical medicine · 2017Review
- International physician survey on management of FOP: a modified Delphi study.Orphanet journal of rare diseases · 2017Article
- Myositis ossificans in children: a review.European journal of orthopaedic surgery & traumatology : orthopedie traumatologie · 2017Article
- Hard targets for a second skeleton: therapeutic horizons for fibrodysplasia ossificans progressiva (FOP).Expert opinion on orphan drugs · 2017Article
- Role of osteoclasts in heterotopic ossification enhanced by fibrodysplasia ossificans progressiva-related activin-like kinase 2 mutation in mice.Journal of bone and mineral metabolism · 2016Article
- High-throughput screening for modulators of ACVR1 transcription: discovery of potential therapeutics for fibrodysplasia ossificans progressiva.Disease models & mechanisms · 2016Article
- Modeling congenital disease and inborn errors of development in Drosophila melanogaster.Disease models & mechanisms · 2016Review
- The Natural History of Flare-Ups in Fibrodysplasia Ossificans Progressiva (FOP): A Comprehensive Global Assessment.Journal of bone and mineral research : the official journal of the American Society for Bone and Mineral Research · 2016Article
Corrections and comments
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Authors and funding
3 authors at 3 institutions in 1 country.
Funding
Abstract
introductionFibrodysplasia ossificans progressiva (FOP) is the most disabling disorder of skeletal metamorphosis in humans and leads to the formation of a second skeleton of heterotopic bone. Presently, there is no effective treatment. AREAS COVERED: In this review, the authors discuss heterozygous activating mutations in Activin receptor A, type I/ Activin-like kinase 2 (ACVR1/ALK2), a bone morphogenetic protein (BMP) type I receptor that are the genetic cause of FOP and reveal a promising pharmacologic target in the BMP signaling pathway. Despite these germline mutations, episodic disease activation is induced by soft tissue injury and resultant inflammatory triggers that are dependent on responding progenitor cells and a tissue microenvironment that supports heterotopic ossification. EXPERT OPINION: Here we review opportunities and challenges for the development of effective therapeutics for FOP. There are many potential approaches that may eventually be used to harness FOP. The long-term treatment of FOP is likely to involve not one, but several concomitant approaches that acknowledge molecular mechanisms involved in the induction and progression of the disease.
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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.