ArticleExpert opinion on orphan drugs2018
Advances in new drug therapies for the management of sickle cell disease.
Article in Expert opinion on orphan drugs, 2018. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 10 papers, 1 of them a synthesis that pooled it.
What it found
Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.
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
10 citing papers in PubMed, 1 synthesis or guideline pooled it, 17 citations in OpenAlex.
- Pooled it
- Adhesion molecules in focus: mechanistic pathways and therapeutic avenues in sickle cell vaso-occlusion - a narrative review.Annals of medicine and surgery (2012) · 2025Review
- Hemoglobin Variants as Targets for Stabilizing Drugs.Molecules (Basel, Switzerland) · 2025Review
- mARC1 Is the Main Contributor to Metabolic Reduction ofJournal of medicinal chemistry · 2024Article
- Review
- Modulating hemoglobin allostery for treatment of sickle cell disease: current progress and intellectual property.Expert opinion on therapeutic patents · 2022Review
- Determinants of hydroxyurea use among doctors, nurses and sickle cell disease patients in Nigeria.PloS one · 2022Article
- What is the future of patient-reported outcomes in sickle-cell disease?Expert review of hematology · 2020Review
- Microfluidics in Sickle Cell Disease Research: State of the Art and a Perspective Beyond the Flow Problem.Frontiers in molecular biosciences · 2020Review
- Emerging disease-modifying therapies for sickle cell disease.Haematologica · 2019Review
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
2 authors at 2 institutions in 1 country.
Funding
Abstract
introductionSickle cell disease (SCD) is an orphan disease in the United States, but is highly prevalent worldwide. Only two drugs, hydroxyurea and L-glutamine, are approved for this disease. With an improved understanding of the pathophysiology of SCD as well as the success of several recently approved drugs for other orphan diseases, there is an increased interest in the development of drugs for SCD. AREAS COVERED: This review summarizes published studies of drug therapies and ongoing trials of novel agents. EXPERT OPINION: The development of drugs with different mechanisms of action offers opportunities for combination and individualized therapy in SCD. In addition to acute pain crisis, the evaluation of other SCD-related complications, exercise capacity, patient reported outcomes and validated surrogate endpoints are necessary to advance drug development. It is important to involve sites in sub-Saharan Africa and India, which have the highest burden of SCD, in trials of novel therapies.
Indexed as
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.