Guidance in social and ethical issues related to clinical, diagnostic care and novel therapies for hereditary neuromuscular rare diseases: "translating" the translational (Q28485060)

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English	Guidance in social and ethical issues related to clinical, diagnostic care and novel therapies for hereditary neuromuscular rare diseases: "translating" the translational	scientific article

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scholarly article

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Guidance in social and ethical issues related to clinical, diagnostic care and novel therapies for hereditary neuromuscular rare diseases: "translating" the translational (English)

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research ethics

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Janbernd Kirschner

8

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Francesco Muntoni

10

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Annemieke Aartsma-Rus

3

object named as

Annemieke Aartsma-Rus

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Thomas Sejersen

object named as

Thomas Sejersen

15

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17

object named as

Volker Straub

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Christoph Rehmann-Sutter

13

object named as

Christoph Rehmann-Sutter

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author name string

Pauline McCormack

1

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Simon Woods

2

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Lynn Hagger

4

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Agnes Herczegfalvi

5

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Emma Heslop

6

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Joseph Irwin

7

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Patrick Moeschen

9

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Marie-Christine Ouillade

11

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Jes Rahbek

12

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Francoise Rouault

14

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Elizabeth Vroom

16

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Kate Bushby

18

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Alessandra Ferlini

19

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language of work or name

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publication date

10 January 2013

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5

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Publication of NIH funded trials registered in ClinicalTrials.gov: cross sectional analysis

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https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3544553

19 March 2017

Informed consent process for patient participation in rare disease registries linked to biorepositories

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https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3544553

19 March 2017

Exon skipping and dystrophin restoration in patients with Duchenne muscular dystrophy after systemic phosphorodiamidate morpholino oligomer treatment: an open-label, phase 2, dose-escalation study

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https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3544553

19 March 2017

Local restoration of dystrophin expression with the morpholino oligomer AVI-4658 in Duchenne muscular dystrophy: a single-blind, placebo-controlled, dose-escalation, proof-of-concept study

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3544553

19 March 2017

Clinical outcome measures for trials in Duchenne muscular dystrophy: report from International Working Group meetings

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3544553

29 September 2017

Antisense oligonucleotide-mediated exon skipping for Duchenne muscular dystrophy: progress and challenges

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29 September 2017

Therapeutic misconception: hope, trust and misconception in paediatric research

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29 September 2017

Phase 1 gene therapy for Duchenne muscular dystrophy using a translational optimized AAV vector

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29 September 2017

Delayed publication of clinical trials in cystic fibrosis

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29 September 2017

Limb girdle muscular dystrophies: update on genetic diagnosis and therapeutic approaches

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29 September 2017

Ex vivo gene transfer and correction for cell-based therapies

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https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3544553

29 September 2017

Systemic administration of PRO051 in Duchenne's muscular dystrophy

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https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3544553

29 September 2017

Ascorbic acid in Charcot-Marie-Tooth disease type 1A (CMT-TRIAAL and CMT-TRAUK): a double-blind randomised trial

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3544553

29 September 2017

Sustained alpha-sarcoglycan gene expression after gene transfer in limb-girdle muscular dystrophy, type 2D.

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3544553

29 September 2017

Creating a European Union framework for actions in the field of rare diseases

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3544553

29 September 2017

Ethics in child and adolescent psychiatric care: An international perspective

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3544553

29 September 2017

Randomized clinical trials in children--ethical and methodological issues

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https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3544553

29 September 2017

The financing of drug trials by pharmaceutical companies and its consequences: part 2: a qualitative, systematic review of the literature on possible influences on authorship, access to trial data, and trial registration and publication

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3544553

29 September 2017

Research ethics. Beyond access vs. protection in trials of innovative therapies

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3544553

29 September 2017

Prevalence of genetic muscle disease in Northern England: in-depth analysis of a muscle clinic population

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3544553

29 September 2017

Ethics and medical research in children

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3544553

29 September 2017

Genetic treatments in muscular dystrophies

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3544553

29 September 2017

Ethical considerations in the interpretation and communication of clinical trial results

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3544553

29 September 2017

The Dutch neuromuscular database CRAMP (Computer Registry of All Myopathies and Polyneuropathies): development and preliminary data

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3544553

29 September 2017

International ethical guidelines for biomedical research involving human subjects.

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3544553

29 September 2017

Early corticosteroid treatment in 4 Duchenne muscular dystrophy patients: 14-year follow-up

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3544553

27 November 2018

Neuromuscular disease: Muscular dystrophy--something new on God's green earth?

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3544553

27 November 2018

Disputing the ethics of research: the challenge from bioethics and patient activism to the interpretation of the Declaration of Helsinki in clinical trials

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3544553

27 November 2018

Access to data in industry-sponsored trials

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https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3544553

27 November 2018

The risks of therapeutic misconception and individual patient (n=1) "trials" in rare diseases such as Duchenne dystrophy.

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https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3544553

27 November 2018

Treatment of infantile Pompe disease with alglucosidase alpha: the UK experience

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3544553

27 November 2018

Seen but not heard? Children in clinical trials

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https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3544553

27 November 2018

Screening for conditions that do not meet the Wilson and Jungner criteria: the case of Duchenne muscular dystrophy.

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3544553

27 November 2018

Trial registers: protecting patients, advancing trust

1 reference

https://pubmed.ncbi.nlm.nih.gov/23330068

12 December 2020

based on heuristic

inferred from PubMed ID database lookup

Evidence-based path to newborn screening for Duchenne muscular dystrophy

1 reference

https://pubmed.ncbi.nlm.nih.gov/23330068

12 December 2020

based on heuristic

inferred from PubMed ID database lookup

Local dystrophin restoration with antisense oligonucleotide PRO051

1 reference

https://pubmed.ncbi.nlm.nih.gov/23330068

12 December 2020

based on heuristic

inferred from PubMed ID database lookup

Best Practice Guidelines on molecular diagnostics in Duchenne/Becker muscular dystrophies

1 reference

https://pubmed.ncbi.nlm.nih.gov/23330068

12 December 2020

based on heuristic

inferred from PubMed ID database lookup

Muscle magnetic resonance imaging in congenital myopathies due to ryanodine receptor type 1 gene mutations

1 reference

https://pubmed.ncbi.nlm.nih.gov/23330068

12 December 2020

based on heuristic

inferred from PubMed ID database lookup

Compliance with mandatory reporting of clinical trial results on ClinicalTrials.gov: cross sectional study

1 reference

https://pubmed.ncbi.nlm.nih.gov/23330068

12 December 2020

based on heuristic

inferred from PubMed ID database lookup

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10.1371/CURRENTS.MD.F90B49429FA814BD26C5B22B13D773EC

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