Muscle biopsies in clinical trials for Duchenne muscular dystrophy – Patients’ and caregivers’ perspective-Reference-Cited by-同舟云学术

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Muscle biopsies in clinical trials for Duchenne muscular dystrophy – Patients’ and caregivers’ perspective

Published:2019-08 Issue:8 Volume:29 Page:576-584
ISSN:0960-8966
Container-title:Neuromuscular Disorders
language:en
Short-container-title:Neuromuscular Disorders

Author:

Verhaart Ingrid E.C.,Johnson Alex,Thakrar Sejal,Vroom Elizabeth,De Angelis Fernanda,Muntoni Francesco,Aartsma-Rus Annemieke M.,Niks Erik H.

Publisher

Elsevier BV

Subject

Genetics (clinical),Neurology (clinical),Neurology,Pediatrics, Perinatology and Child Health

Reference30 articles.

1. The muscular dystrophies;Emery;Lancet,2002

2. Dystrophin: the protein product of the Duchenne muscular dystrophy locus;Hoffman;Cell,1987

3. Function and genetics of dystrophin and dystrophin-related proteins in muscle;Blake;Physiol Rev,2002

4. An overview of recent therapeutics advances for Duchenne muscular dystrophy;Mah;Methods Mol Biol,2018

5. 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;Kinali;Lancet Neurol,2009

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1. Draft Guidance for Industry Duchenne Muscular Dystrophy, Becker Muscular Dystrophy, and Related Dystrophinopathies – Developing Potential Treatments for the Entire Spectrum of Disease;Journal of Neuromuscular Diseases;2024-03-05

2. Facioscapulohumeral Muscular Dystrophy European Patient Survey: Assessing Patient Reported Disease Burden and Preferences in Clinical Trial Participation;Journal of Neuromuscular Diseases;2024-03-05

3. The Dilemma of Choice for Duchenne Patients Eligible for Exon 51 Skipping The European Experience;Journal of Neuromuscular Diseases;2023-05-02

4. Assessment of the burden of outpatient clinic and MRI-guided needle muscle biopsies as reported by patients with facioscapulohumeral muscular dystrophy;Neuromuscular Disorders;2023-05

5. Perspectives on Participation in Clinical Trials Among Individuals With Pain, Depression, and/or Anxiety: An ACTTION Scoping Review;The Journal of Pain;2023-01

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