Report of a TREAT-NMD/World Duchenne Organisation Meeting on Dystrophin Quantification Methodology (Q64041594)

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17 April 2020

title

Report of a TREAT-NMD/World Duchenne Organisation Meeting on Dystrophin Quantification Methodology (English)

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17 April 2020

author

Annemieke Aartsma-Rus

1

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17 April 2020

Marisol Montolio

7

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Virginia Arechavala-Gomeza

17 April 2020

15

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17 April 2020

13

Eric P Hoffman

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17 April 2020

10

Judith Van Deutekom

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17 April 2020

14

Francesco Muntoni

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17 April 2020

author name string

Jennifer Morgan

2

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17 April 2020

Pallavi Lonkar

3

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17 April 2020

Hendrik Neubert

4

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17 April 2020

Jane Owens

5

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17 April 2020

Michael Binks

6

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17 April 2020

Rahul Phadke

8

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17 April 2020

Nicole Datson

9

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17 April 2020

Glenn E Morris

11

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17 April 2020

V Ashutosh Rao

12

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17 April 2020

workshop participants

16

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17 April 2020

language of work or name

English

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publication date

1 January 2019

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Journal of neuromuscular diseases

17 April 2020

published in

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17 April 2020

volume

6

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17 April 2020

issue

1

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17 April 2020

page(s)

147-159

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Creative Commons Attribution-NonCommercial 4.0 International

17 April 2020

copyright license

start time

30 January 2019

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April 2022 Public Data File from Crossref

copyright status

copyrighted

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cites work

Monoclonal antibodies against defined regions of the muscular dystrophy protein, dystrophin

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12 December 2020

Exploring the molecular basis for variability among patients with Becker muscular dystrophy: dystrophin gene and protein studies

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Improved diagnosis of Becker muscular dystrophy by dystrophin testing

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The complete sequence of dystrophin predicts a rod-shaped cytoskeletal protein

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The N-terminal half of dystrophin is protected from proteolysis in situ

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Massive idiosyncratic exon skipping corrects the nonsense mutation in dystrophic mouse muscle and produces functional revertant fibers by clonal expansion

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Modular flexibility of dystrophin: implications for gene therapy of Duchenne muscular dystrophy

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The phenotype of limb-girdle muscular dystrophy type 2I

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An open letter to our readers on the use of antibodies

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Entries in the Leiden Duchenne muscular dystrophy mutation database: an overview of mutation types and paradoxical cases that confirm the reading-frame rule

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12 December 2020

Dystrophin levels as low as 30% are sufficient to avoid muscular dystrophy in the human

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A guide to the perplexed on the specificity of antibodies

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Immunohistological intensity measurements as a tool to assess sarcolemma-associated protein expression

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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

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12 December 2020

Revertant fibres and dystrophin traces in Duchenne muscular dystrophy: Implication for clinical trials

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12 December 2020

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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12 December 2020

Dystrophin quantification and clinical correlations in Becker muscular dystrophy: implications for clinical trials

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12 December 2020

Quantification of dystrophin immunofluorescence in dystrophinopathy muscle specimens

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12 December 2020

Assessment of the structural and functional impact of in-frame mutations of the DMD gene, using the tools included in the eDystrophin online database

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12 December 2020

Proteomic analysis reveals new cardiac-specific dystrophin-associated proteins

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12 December 2020

Genetic and clinical specificity of 26 symptomatic carriers for dystrophinopathies at pediatric age

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12 December 2020

Low dystrophin levels increase survival and improve muscle pathology and function in dystrophin/utrophin double-knockout mice

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12 December 2020

Clinical characterisation of Becker muscular dystrophy patients predicts favourable outcome in exon-skipping therapy.

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12 December 2020

Eteplirsen for the treatment of Duchenne muscular dystrophy

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12 December 2020

Biochemical characterization of patients with in-frame or out-of-frame DMD deletions pertinent to exon 44 or 45 skipping

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12 December 2020

Dystrophin levels and clinical severity in Becker muscular dystrophy patients.

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12 December 2020

Principles of analytic validation of immunohistochemical assays: Guideline from the College of American Pathologists Pathology and Laboratory Quality Center

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12 December 2020

Exon skipping restores dystrophin expression, but fails to prevent disease progression in later stage dystrophic dko mice

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12 December 2020

Controls for immunohistochemistry: the Histochemical Society's standards of practice for validation of immunohistochemical assays

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12 December 2020

Safety and efficacy of drisapersen for the treatment of Duchenne muscular dystrophy (DEMAND II): an exploratory, randomised, placebo-controlled phase 2 study

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12 December 2020

A sensitive, reproducible and objective immunofluorescence analysis method of dystrophin in individual fibers in samples from patients with duchenne muscular dystrophy

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12 December 2020

Long term natural history data in ambulant boys with Duchenne muscular dystrophy: 36-month changes.

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12 December 2020

Dystrophin quantification: Biological and translational research implications

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12 December 2020

How much dystrophin is enough: the physiological consequences of different levels of dystrophin in the mdx mouse

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Large-scale serum protein biomarker discovery in Duchenne muscular dystrophy

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12 December 2020

Immune-mediated pathology in Duchenne muscular dystrophy

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12 December 2020

TNF-α-Induced microRNAs Control Dystrophin Expression in Becker Muscular Dystrophy

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Quantitative Analysis of Human Neonatal Fc Receptor (FcRn) Tissue Expression in Transgenic Mice by Online Peptide Immuno-Affinity LC-HRMS.

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Stakeholder cooperation to overcome challenges in orphan medicine development: the example of Duchenne muscular dystrophy.

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Long-term microdystrophin gene therapy is effective in a canine model of Duchenne muscular dystrophy.

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Myoblots: dystrophin quantification by in-cell western assay for a streamlined development of Duchenne muscular dystrophy (DMD) treatments

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A randomized placebo-controlled phase 3 trial of an antisense oligonucleotide, drisapersen, in Duchenne muscular dystrophy

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Micro-dystrophin gene therapy goes systemic in Duchenne muscular dystrophy patients

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12 December 2020

Dystrophin's central domain forms a complex filament that becomes disorganized by in-frame deletions.

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12 December 2020

A novel high-throughput immunofluorescence analysis method for quantifying dystrophin intensity in entire transverse sections of Duchenne muscular dystrophy muscle biopsy samples.

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12 December 2020

Low dystrophin levels are insufficient to normalize the neuromuscular synaptic abnormalities of mdx mice

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Use of capillary Western immunoassay (Wes) for quantification of dystrophin levels in skeletal muscle of healthy controls and individuals with Becker and Duchenne muscular dystrophy.

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Why dystrophin quantification is key in the eteplirsen saga

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Systemic AAV Micro-dystrophin Gene Therapy for Duchenne Muscular Dystrophy

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12 December 2020

Identifiers

DOI

10.3233/JND-180357

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17 April 2020

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17 April 2020

PubMed publication ID

30614809

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