ArticleNature biotechnology2019
What compassionate use means for gene therapies.
Article in Nature biotechnology, 2019. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 12 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
12 citing papers in PubMed, 1 synthesis or guideline pooled it.
- Pooled it
- Roadmap for direct and indirect translation of optogenetics into discoveries and therapies for humans.Nature neuroscience · 2025Review
- Augmenting Treatment Arms With External Data Through Propensity-Score Weighted Power Priors: An Application in Expanded Access.Statistics in medicine · 2025Article
- A conceptual model and practical guidance for the development, administration, and evaluation of individualized therapies.Frontiers in medicine · 2025Article
- Regulating clinical trials in a resource-limited setting during the Ebola public health emergency in Sierra Leone.Trials · 2022Article
- Real-world data from expanded access programmes in health technology assessments: a review of NICE technology appraisals.BMJ open · 2022Review
- Early access provision: Awareness, educational needs and opportunities to improve oncology patients' access to care.Frontiers in oncology · 2022Article
- Generating Evidence from Expanded Access Use of Rare Disease Medicines: Challenges and Recommendations.Frontiers in pharmacology · 2022Article
- Prospect of compassionate use in China from remdesivir.Zhong nan da xue xue bao. Yi xue ban = Journal of Central South University. Medical sciences · 2021Article
- Financial considerations in expanded access policy for gene therapies: A tough nut to crack?Molecular therapy : the journal of the American Society of Gene Therapy · 2021Article
- Gene therapy companies have an ethical obligation to develop expanded access policies.Molecular therapy : the journal of the American Society of Gene Therapy · 2021Article
- Expanded Access as a source of real-world data: An overview of FDA and EMA approvals.British journal of clinical pharmacology · 2020Article
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
7 authors.
Funding
No grant is acknowledged in the PubMed record.
Abstract
PubMed holds no abstract for this paper.
Indexed as
Identifiers
30940936What 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.