ArticleAnnals of neurology2022
Ethical Perspectives on Treatment Options with Spinal Muscular Atrophy Patients.
Article in Annals of neurology, 2022. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 19 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
19 citing papers in PubMed, 41 citations in OpenAlex.
- Addressing the Global Disparities in Access to Treatment in Spinal Muscular Atrophy.Muscle & nerve · 2026Review
- Consensus for the Diagnosis and Treatment of Patients With Spinal Muscular Atrophy (SMA) in Latin America.Revista de neurologia · 2026Article
- "The Most Difficult Conversations We Have": Resident Experiences With Pediatric Neuroprognostication.Annals of the Child Neurology Society · 2026Article
- Do the math: modernizing the FDA's evidence paradigm for precision medicine.The Journal of clinical investigation · 2026Article
- Liver Steatosis in Induced Hepatocytes From Carriers of Spinal Muscular Atrophy.Muscle & nerve · 2026Article
- Preventing spinal muscular atrophy through the national premarital screening program in Türkiye: an economic comparison with treatment costs.Turkish journal of medical sciences · 2026Article
- A neuromuscular clinician's primer on machine learning.Journal of neuromuscular diseases · 2026Review
- Disease characteristics and treatment status of genetically confirmed spinal muscular atrophy patients: a cross-sectional survey in China.BMC neurology · 2025Article
- Safety of Risdiplam in Japanese Patients with Spinal Muscular Atrophy: A 12‑Month Interim Analysis of a Postmarketing Surveillance Study.Neurology and therapy · 2025Article
- Editorial - Patient perspectives on new therapies for genetic diseases.Medizinische Genetik : Mitteilungsblatt des Berufsverbandes Medizinische Genetik e.V · 2025Article
- Current clinical applications of AAV-mediated gene therapy.Molecular therapy : the journal of the American Society of Gene Therapy · 2025Review
- Risdiplam in Adult Patients With 5q Spinal Muscular Atrophy: A Single-Center Longitudinal Study.Muscle & nerve · 2025Observational
- Patients' Perceptions of Nusinersen Effects According to Their Responder Status.Journal of clinical medicine · 2024Article
- Life-Saving Treatments for Spinal Muscular Atrophy: Global Access and Availability.Neurology. Clinical practice · 2024Article
- Gene therapy-based strategies for spinal muscular atrophy-an Asia-Pacific perspective.Molecular and cellular pediatrics · 2023Review
- Identification of potential pathways and biomarkers linked to progression in ALS.Annals of clinical and translational neurology · 2023Article
- The diagnosis communication process in spinal muscular atrophy: A cross-cutting view of the new challenges facing the therapeutic era.Genetics in medicine open · 2023Review
- Onasemnogene Abeparvovec: A Review in Spinal Muscular Atrophy.CNS drugs · 2022Review
- Spinal muscular atrophy.Nature reviews. Disease primers · 2022Review
Corrections and comments
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Authors and funding
4 authors at 4 institutions in 3 countries.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Since 2016, 3 innovative therapies for spinal muscular atrophy (SMA) have changed the face of the disease. Although these therapies often result in remarkable improvements in infants and children, benefits in adults are modest and treatment is not curative. Concerns have been raised about the enormous costs of these medications, the ultimate burden to taxpayers, and the costs to society of withholding treatments and sacrificing or disadvantaging some individuals. Physicians are best positioned to serve our patients by carefully considering the costs, benefits, implications for quality of life (QOL), and the interplay of these factors within the framework of core ethical principles that guide clinical care. ANN NEUROL 2022;91:305-316.
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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.