ReviewGene therapy2017
New treatments for serious conditions: ethical implications.
Review in Gene therapy, 2017. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 13 papers.
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
13 citing papers in PubMed, 37 citations in OpenAlex.
- A review of methods and software for polygenic risk score analysis.PeerJ. Computer science · 2025Article
- Ethical implications of developing RNA-based therapies for cardiovascular disorders.Frontiers in bioengineering and biotechnology · 2024Review
- A qualitative study on the views of experts on the social impact of the high-priced orphan drug nusinersen.Exploratory research in clinical and social pharmacy · 2023Article
- Do we always need to treat patients with spinal muscular atrophy? A personal view and experience.Orphanet journal of rare diseases · 2021Article
- Assessing the Value of Nusinersen for Spinal Muscular Atrophy: A Comparative Analysis of Reimbursement Submission and Appraisal in European Countries.Frontiers in pharmacology · 2021Review
- Effects of nusinersen after one year of treatment in 123 children with SMA type 1 or 2: a French real-life observational study.Orphanet journal of rare diseases · 2020Observational
- Review
- Palliative Care in SMA Type 1: A Prospective Multicenter French Study Based on Parents' Reports.Frontiers in pediatrics · 2020Article
- Perspectives in genetic counseling for spinal muscular atrophy in the new therapeutic era: early pre-symptomatic intervention and test in minors.European journal of human genetics : EJHG · 2019Review
- Recent developments in genetic/genomic medicine.Clinical science (London, England : 1979) · 2019Review
- Gene Therapy for Spinal Muscular Atrophy: An Emerging Treatment Option for a Devastating Disease.Journal of managed care & specialty pharmacy · 2018Review
- Limited maximal mouth opening in patients with spinal muscular atrophy complicates endotracheal intubation: An observational study.European journal of anaesthesiology · 2018Observational
- Advances in therapy for spinal muscular atrophy: promises and challenges.Nature reviews. Neurology · 2018Review
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 1 institution in 1 country.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Approval of Spinraza (nusinersen) for treatment of spinal muscular atrophy prompts consideration of a number of ethical issues that arise whenever a new treatment is proposed for a serious condition, especially one that is rare and can devastatingly affect children. Patients, families, clinicians, researchers, institutions and policymakers all must take account of the ways that newly available treatments affect informed and shared decision-making about therapeutic and research options. The issues to consider include: addressing what is still uncertain and unknown; the possibility that potential benefits will be exaggerated and potential harms underemphasized in the media, by advocacy organizations, and in consent forms and processes; the high cost of many novel drugs and biologics; the effects of including conditions of variable phenotype in state-mandated newborn screening panels; and how new treatments can change the standard of care, altering what is and is not known about a disorder and posing challenges for decision-making at both individual and policy levels. The good news that Spinraza brings thus requires additional attention to its ethical and policy implications, to improve counseling and shared decision-making about treatment and research options for patients and all involved in their care.
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.