A paper a day Archives • World Duchenne

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#apaperaday: Gene Therapy for Duchenne Muscular Dystrophy: Unlocking the Opportunities in Countries in the Middle East and Beyond

Gene Therapy for Duchenne Muscular Dystrophy: Unlocking the Opportunities in Countries in the Middle East and Beyond

#apaperaday: Perspectives on the advances in the pharmacotherapeutic management of Duchenne muscular dystrophy

Perspectives on the advances in the pharmacotherapeutic management of Duchenne muscular dystrophy

#apaperaday: Mineralocorticoid receptor antagonists and glucocorticoids differentially affect skeletal muscle inflammation and pathology in muscular dystrophy

Mineralocorticoid receptor antagonists and glucocorticoids differentially affect skeletal muscle inflammation and pathology in muscular dystrophy

#apaperaday: Patient-led development of digital endpoints and the use of computer vision analysis in assessment of motor function in rare diseases

Patient-led development of digital endpoints and the use of computer vision analysis in assessment of motor function in rare diseases

#apaperaday: A first-in-human phase I/IIa gene transfer clinical trial for Duchenne muscular dystrophy

A first-in-human phase I/IIa gene transfer clinical trial for Duchenne muscular dystrophy

#apaperaday: Newborn screening for Duchenne muscular dystrophy-early detection and diagnostic algorithm for female carriers of Duchenne muscular dystrophy

Newborn screening for Duchenne muscular dystrophy-early detection and diagnostic algorithm for female carriers of Duchenne muscular dystrophy

#apaperaday: Generation of two induced pluripotent stem cell (iPSC) lines from patients with Duchenne muscular dystrophy

Generation of two induced pluripotent stem cell (iPSC) lines from patients with Duchenne muscular dystrophy

#apaperaday: Cognitive and behavioral functioning in two neurogenetic disorders: Duchenne muscular dystrophy and Neurofibromatosis type 1

Cognitive and behavioral functioning in two neurogenetic disorders: Duchenne muscular dystrophy and Neurofibromatosis type 1

#apaperaday: A cell-penetrating peptide enhances delivery and efficacy of phosphorodiamidate morpholino oligomers in mdx mice

A cell-penetrating peptide enhances delivery and efficacy of phosphorodiamidate morpholino oligomers in mdx mice

#apaperaday: Semirational bioengineering of AAV vectors with increased potency and specificity for systemic gene therapy of muscle disorders

Semirational bioengineering of AAV vectors with increased potency and specificity for systemic gene therapy of muscle disorders

#apaperaday: Siblings’ life aspirations in the context of Duchenne muscular dystrophy

Siblings’ life aspirations in the context of Duchenne muscular dystrophy

#apaperaday: Exploring the Therapeutic Potential of Ectoine in Duchenne Muscular Dystrophy

Exploring the Therapeutic Potential of Ectoine in Duchenne Muscular Dystrophy

#apaperaday: RNA-seq analysis, targeted long-read sequencing and in silico prediction to unravel pathogenic intronic events and complicated splicing abnormalities in dystrophinopathy

RNA-seq analysis, targeted long-read sequencing and in silico prediction to unravel pathogenic intronic events and complicated splicing abnormalities in dystrophinopathy

#apaperaday: The neurocognitive profile of adults with Becker muscular dystrophy in the Netherlands

The neurocognitive profile of adults with Becker muscular dystrophy in the Netherlands

#apaperaday: Prime Editing Permits the Introduction of Specific Mutations in the Gene Responsible for Duchenne Muscular Dystrophy

Prime Editing Permits the Introduction of Specific Mutations in the Gene Responsible for Duchenne Muscular Dystrophy.

#apaperaday: Longitudinal assessment of blood-borne musculoskeletal disease biomarkers in the DE50-MD dog model of Duchenne muscular dystrophy

Longitudinal assessment of blood-borne musculoskeletal disease biomarkers in the DE50-MD dog model of Duchenne muscular dystrophy

#apaperaday: The gRNA Vector Level Determines the Outcome of Systemic AAV CRISPR Therapy for Duchenne Muscular Dystrophy

The gRNA Vector Level Determines the Outcome of Systemic AAV CRISPR Therapy for Duchenne Muscular Dystrophy

#apaperaday: Factors associated with the health-related quality of life among people with Duchenne muscular dystrophy

Factors associated with the health-related quality of life among people with Duchenne muscular dystrophy

#apaperaday: Exercise Training in Duchenne Muscular Dystrophy: A Systematic Review and Meta-Analysis

Exercise Training in Duchenne Muscular Dystrophy: A Systematic Review and Meta-Analysis

#apaperaday: Antioxidant effects of bis-indole alkaloid indigo and related signaling pathways in the experimental model of Duchenne muscular dystrophy

Antioxidant effects of bis-indole alkaloid indigo and related signaling pathways in the experimental model of Duchenne muscular dystrophy

Facilitating [corrected] More Efficient Negotiations for Innovative Therapies- A Value-Based Negotiation Framework

#apaperaday: Facilitating More Efficient Negotiations for Innovative Therapies

Facilitating More Efficient Negotiations for Innovative Therapies: A Value-Based Negotiation Framework

#apaperaday: Pig models for Duchenne muscular dystrophy

Pig models for Duchenne muscular dystrophy - from disease mechanisms to validation of new diagnostic and therapeutic concepts.

#apaperaday: Do NGS-based techniques represent a first-line testing in suspected Duchenne muscular dystrophy?

Do NGS-based techniques represent a first-line testing in suspected Duchenne muscular dystrophy?

#apaperaday: Transplantation of human iPSC-derived muscle stem cells in the diaphragm of Duchenne muscular dystrophy model mice

Transplantation of human iPSC-derived muscle stem cells in the diaphragm of Duchenne muscular dystrophy model mice

#apaperaday: Long-term maintenance of dystrophin expression and resistance to injury of skeletal muscle in gene edited DMD mice

Long-term maintenance of dystrophin expression and resistance to injury of skeletal muscle in gene edited DMD mice

#apaperaday: Functional outcome measures in young, steroid-naïve boys with Duchenne muscular dystrophy

Functional outcome measures in young, steroid-naïve boys with Duchenne muscular dystrophy

#apaperaday: mRNA-mediated delivery of gene editing tools to human primary muscle stem cells

mRNA-mediated delivery of gene editing tools to human primary muscle stem cells

#apaperaday: Successful bone marrow transplantation in a patient with Diamond-Blackfan anemia with co-existing Duchenne muscular dystrophy: a case report

Successful bone marrow transplantation in a patient with Diamond-Blackfan anemia with co-existing Duchenne muscular dystrophy: a case report

#apaperaday: Prognostic indicators of disease progression in Duchenne muscular dystrophy: A literature review and evidence synthesis

Prognostic indicators of disease progression in Duchenne muscular dystrophy: A literature review and evidence synthesis

#apaperaday: Long-Term Functional Efficacy and Safety of Viltolarsen in Patients with Duchenne Muscular Dystrophy

Long-Term Functional Efficacy and Safety of Viltolarsen in Patients with Duchenne Muscular Dystrophy

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#apaperaday: Gene Therapy for Duchenne Muscular Dystrophy: Unlocking the Opportunities in Countries in the Middle East and Beyond

#apaperaday: Perspectives on the advances in the pharmacotherapeutic management of Duchenne muscular dystrophy

#apaperaday: Mineralocorticoid receptor antagonists and glucocorticoids differentially affect skeletal muscle inflammation and pathology in muscular dystrophy

#apaperaday: Patient-led development of digital endpoints and the use of computer vision analysis in assessment of motor function in rare diseases

#apaperaday: A first-in-human phase I/IIa gene transfer clinical trial for Duchenne muscular dystrophy

#apaperaday: Newborn screening for Duchenne muscular dystrophy-early detection and diagnostic algorithm for female carriers of Duchenne muscular dystrophy

#apaperaday: Generation of two induced pluripotent stem cell (iPSC) lines from patients with Duchenne muscular dystrophy

#apaperaday: Cognitive and behavioral functioning in two neurogenetic disorders: Duchenne muscular dystrophy and Neurofibromatosis type 1

#apaperaday: A cell-penetrating peptide enhances delivery and efficacy of phosphorodiamidate morpholino oligomers in mdx mice

#apaperaday: Semirational bioengineering of AAV vectors with increased potency and specificity for systemic gene therapy of muscle disorders

#apaperaday: Siblings’ life aspirations in the context of Duchenne muscular dystrophy

#apaperaday: Exploring the Therapeutic Potential of Ectoine in Duchenne Muscular Dystrophy

#apaperaday: RNA-seq analysis, targeted long-read sequencing and in silico prediction to unravel pathogenic intronic events and complicated splicing abnormalities in dystrophinopathy

#apaperaday: The neurocognitive profile of adults with Becker muscular dystrophy in the Netherlands

#apaperaday: Prime Editing Permits the Introduction of Specific Mutations in the Gene Responsible for Duchenne Muscular Dystrophy

#apaperaday: Longitudinal assessment of blood-borne musculoskeletal disease biomarkers in the DE50-MD dog model of Duchenne muscular dystrophy

#apaperaday: The gRNA Vector Level Determines the Outcome of Systemic AAV CRISPR Therapy for Duchenne Muscular Dystrophy

#apaperaday: Factors associated with the health-related quality of life among people with Duchenne muscular dystrophy

#apaperaday: Exercise Training in Duchenne Muscular Dystrophy: A Systematic Review and Meta-Analysis

#apaperaday: Antioxidant effects of bis-indole alkaloid indigo and related signaling pathways in the experimental model of Duchenne muscular dystrophy

#apaperaday: Facilitating More Efficient Negotiations for Innovative Therapies

#apaperaday: Pig models for Duchenne muscular dystrophy

#apaperaday: Do NGS-based techniques represent a first-line testing in suspected Duchenne muscular dystrophy?

#apaperaday: Transplantation of human iPSC-derived muscle stem cells in the diaphragm of Duchenne muscular dystrophy model mice

#apaperaday: Long-term maintenance of dystrophin expression and resistance to injury of skeletal muscle in gene edited DMD mice

#apaperaday: Functional outcome measures in young, steroid-naïve boys with Duchenne muscular dystrophy

#apaperaday: mRNA-mediated delivery of gene editing tools to human primary muscle stem cells

#apaperaday: Successful bone marrow transplantation in a patient with Diamond-Blackfan anemia with co-existing Duchenne muscular dystrophy: a case report

#apaperaday: Prognostic indicators of disease progression in Duchenne muscular dystrophy: A literature review and evidence synthesis

#apaperaday: Long-Term Functional Efficacy and Safety of Viltolarsen in Patients with Duchenne Muscular Dystrophy

World Duchenne Organization announces Accredited Duchenne Centers Program

Official WDAD2022 Event: Women and Duchenne

World Duchenne Organization Leadership Award for Professor Muntoni