Clinical outcome measures for trials in Duchenne muscular dystrophy: report from International Working Group meetings (Q30514152)

11 August 2017

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

1 reference

Duchenne muscular dystrophy

11 August 2017

1 reference

Kate Bushby

1

1 reference

PMCID

3357954

11 August 2017

Edward Connor

series ordinal

2

1 reference

11 August 2017

1 September 2011

1 reference

11 August 2017

Clinical investigation

1 reference

11 August 2017

1

1 reference

11 August 2017

9

1 reference

11 August 2017

1217-1235

1 reference

Nonsense mutation-associated Becker muscular dystrophy: interplay between exon definition and splicing regulatory elements within the DMD gene

11 August 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3357954

Restoring dystrophin expression in duchenne muscular dystrophy muscle progress in exon skipping and stop codon read through

20 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3357954

Moving towards meaningful measurement: Rasch analysis of the North Star Ambulatory Assessment in Duchenne muscular dystrophy

20 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3357954

Treatment of Duchenne muscular dystrophy with ciclosporin A: a randomised, double-blind, placebo-controlled multicentre trial

20 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3357954

Clinical and genetic characterization of manifesting carriers of DMD mutations

20 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3357954

Analysis of dystrophin deletion mutations predicts age of cardiomyopathy onset in becker muscular dystrophy

20 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3357954

Mutational spectrum of DMD mutations in dystrophinopathy patients: application of modern diagnostic techniques to a large cohort

20 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3357954

DMD Trp3X nonsense mutation associated with a founder effect in North American families with mild Becker muscular dystrophy

20 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3357954

Reliability of the North Star Ambulatory Assessment in a multicentric setting.

20 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3357954

DMD exon 1 truncating point mutations: amelioration of phenotype by alternative translation initiation in exon 6.

20 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3357954

Rating scales as outcome measures for clinical trials in neurology: problems, solutions, and recommendations

20 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3357954

Readthrough of dystrophin stop codon mutations induced by aminoglycosides

20 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3357954

Rapid direct sequence analysis of the dystrophin gene

20 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3357954

Profiles of neuromuscular diseases. Duchenne muscular dystrophy.

20 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3357954

North Star Ambulatory Assessment, 6-minute walk test and timed items in ambulant boys with Duchenne muscular dystrophy

20 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3357954

The 6-minute walk test as a new outcome measure in Duchenne muscular dystrophy.

26 November 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3357954

Monitoring changes and predicting loss of ambulation in Duchenne muscular dystrophy with the Motor Function Measure

26 November 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3357954

Reliable surrogate outcome measures in multicenter clinical trials of Duchenne muscular dystrophy.

26 November 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3357954

Physical capacity in non-ambulatory people with Duchenne muscular dystrophy or spinal muscular atrophy: a longitudinal study.

26 November 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3357954

Clinical evaluator reliability for quantitative and manual muscle testing measures of strength in children.

26 November 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3357954

The development of antisense oligonucleotide therapies for Duchenne muscular dystrophy: report on a TREAT-NMD workshop hosted by the European Medicines Agency (EMA), on September 25th 2009

26 November 2018

1 reference

12 December 2020

DMD pseudoexon mutations: splicing efficiency, phenotype, and potential therapy

1 reference

12 December 2020

Clinical trial in Duchenne dystrophy. I. The design of the protocol

1 reference

12 December 2020

Duplications in the DMD gene

1 reference

12 December 2020

Improved molecular diagnosis of dystrophinopathies in an unselected clinical cohort

1 reference

12 December 2020

Management of progressive muscular dystrophy in childhood

1 reference

12 December 2020

Clinical trials in Duchenne dystrophy. Standardization and reliability of evaluation procedures

1 reference

12 December 2020

A motor function measure for neuromuscular diseases. Construction and validation study

1 reference

12 December 2020

Validity of the EK scale: a functional assessment of non-ambulatory individuals with Duchenne muscular dystrophy or spinal muscular atrophy

1 reference

12 December 2020

Indicators of need for mechanical ventilation in Duchenne muscular dystrophy and spinal muscular atrophy

1 reference

12 December 2020

Identifiers

DOI

10.4155/CLI.11.113

1 reference

11 August 2017

1 reference

11 August 2017

1 reference

PMCID

3357954