1
40
2
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Dublin Core
The Dublin Core metadata element set is common to all Omeka records, including items, files, and collections. For more information see, http://dublincore.org/documents/dces/.
Title
A name given to the resource
August 2022 List
Text
A resource consisting primarily of words for reading. Examples include books, letters, dissertations, poems, newspapers, articles, archives of mailing lists. Note that facsimiles or images of texts are still of the genre Text.
Citation List Month
August 2022 List
URL Address
<a href="http://doi.org/10.1038/s41436-021-01245-3" target="_blank" rel="noreferrer noopener">http://doi.org/10.1038/s41436-021-01245-3</a>
Dublin Core
The Dublin Core metadata element set is common to all Omeka records, including items, files, and collections. For more information see, http://dublincore.org/documents/dces/.
Title
A name given to the resource
Ethical challenges for a new generation of early-phase pediatric gene therapy trials
Publisher
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Genetics in Medicine
Date
A point or period of time associated with an event in the lifecycle of the resource
2021
Subject
The topic of the resource
pediatrics; ethics; human; review; clinical trial (topic); patient selection; patient engagement; risk benefit analysis; gene therapy; giant axonal neuropathy
Creator
An entity primarily responsible for making the resource
Iyer AA; Saade D; Bharucha-Goebel D; Foley AR; Averion GM; Paredes E; Gray S; Bonnemann CG; Grady C; Hendriks S; Rid A
Description
An account of the resource
After decades of setbacks, gene therapy (GT) is experiencing major breakthroughs. Five GTs have received US regulatory approval since 2017, and over 900 others are currently in development. Many of these GTs target rare pediatric diseases that are severely life-limiting, given a lack of effective treatments. As these GTs enter early-phase clinical trials, specific ethical challenges remain unresolved in three domains: evaluating risks and potential benefits, selecting participants fairly, and engaging with patient communities. Drawing on our experience as clinical investigators, basic scientists, and bioethicists involved in a first-in-human GT trial for an ultrarare pediatric disease, we analyze these ethical challenges and offer points to consider for future GT trials. Copyright © 2021, This is a U.S. government work and not under copyright protection in the U.S.; foreign copyright protection may apply.
Identifier
An unambiguous reference to the resource within a given context
<a href="http://doi.org/10.1038/s41436-021-01245-3" target="_blank" rel="noreferrer noopener">10.1038/s41436-021-01245-3</a>
Rights
Information about rights held in and over the resource
Article information provided for research and reference use only. PedPalASCNET does not hold any rights over the resource listed here. All rights are retained by the journal listed under publisher and/or the creator(s).
2021
August 2022 List
Averion GM
Bharucha-Goebel D
Bonnemann CG
Clinical Trial (topic)
Ethics
Foley AR
Gene Therapy
Genetics in Medicine
giant axonal neuropathy
Grady C
Gray S
Hendriks S
Human
Iyer AA
Paredes E
patient engagement
Patient Selection
Pediatrics
Review
Rid A
Risk Benefit Analysis
Saade D
-
Text
A resource consisting primarily of words for reading. Examples include books, letters, dissertations, poems, newspapers, articles, archives of mailing lists. Note that facsimiles or images of texts are still of the genre Text.
Citation List Month
Backlog
URL Address
<a href="http://doi.org/10.1542/peds.2012-0068" target="_blank" rel="noreferrer">http://doi.org/10.1542/peds.2012-0068</a>
Dublin Core
The Dublin Core metadata element set is common to all Omeka records, including items, files, and collections. For more information see, http://dublincore.org/documents/dces/.
Title
A name given to the resource
Views of adolescents and parents on pediatric research without the potential for clinical benefit
Publisher
An entity responsible for making the resource available
Pediatrics
Date
A point or period of time associated with an event in the lifecycle of the resource
2012
Subject
The topic of the resource
Female; Humans; Male; Adolescent Psychology; Adult; Attitude to Health; Questionnaires; Motivation; Altruism; adolescent; Risk-Taking; Parents/psychology; Nontherapeutic Human Experimentation; Research Subjects/psychology; Benefits of PPC
Creator
An entity primarily responsible for making the resource
Wendler D; Abdoler E; Wiener L; Grady C
Identifier
An unambiguous reference to the resource within a given context
<a href="http://doi.org/10.1542/peds.2012-0068" target="_blank" rel="noreferrer">10.1542/peds.2012-0068</a>
Rights
Information about rights held in and over the resource
Article information provided for research and reference use only. PedPalASCNET does not hold any rights over the resource listed here. All rights are retained by the journal listed under publisher and/or the creator(s).
Type
The nature or genre of the resource
Journal Article
2012
Abdoler E
Adolescent
Adolescent Psychology
Adult
Altruism
Attitude To Health
Backlog
Benefits of PPC
Female
Grady C
Humans
Journal Article
Male
Motivation
Nontherapeutic Human Experimentation
Parents/psychology
Pediatrics
Questionnaires
Research Subjects/psychology
Risk-Taking
Wendler D
Wiener L