Paper a day Archives • World Duchenne

Showing all 30 results

#apaperaday: N-of-1 Healthcare: Challenges and Prospects for the Future of Personalized Medicine

N-of-1 Healthcare: Challenges and Prospects for the Future of Personalized Medicine

#apaperaday: The X-linked Becker muscular dystrophy (bmx) mouse models Becker muscular dystrophy via deletion of murine dystrophin exons 45–47

The X-linked Becker muscular dystrophy (bmx) mouse models Becker muscular dystrophy via deletion of murine dystrophin exons 45–47

#apaperaday: Microdystrophin Gene Addition Significantly Improves Muscle Functionality and Diaphragm Muscle Histopathology in a Fibrotic Mouse Model of Duchenne Muscular Dystrophy

Microdystrophin Gene Addition Significantly Improves Muscle Functionality and Diaphragm Muscle Histopathology in a Fibrotic Mouse Model of Duchenne Muscular Dystrophy

#apaperaday: Micro-dystrophin gene therapy demonstrates long-term cardiac efficacy in a severe Duchenne muscular dystrophy model

Micro-dystrophin gene therapy demonstrates long-term cardiac efficacy in a severe Duchenne muscular dystrophy model

#apaperaday: Unexpected Death of a Duchenne Muscular Dystrophy Patient in an N-of-1 Trial of rAAV9-delivered CRISPR-transactivator

Unexpected Death of a Duchenne Muscular Dystrophy Patient in an N-of-1 Trial of rAAV9-delivered CRISPR-transactivator

#apaperaday: A Mixed-Method Study Exploring Patient-Experienced and Caregiver-Reported Benefits and Side Effects of Corticosteroid Use in Duchenne Muscular Dystrophy

A Mixed-Method Study Exploring Patient-Experienced and Caregiver-Reported Benefits and Side Effects of Corticosteroid Use in Duchenne Muscular Dystrophy

#apaperaday: Delivery challenges for CRISPR-Cas9 genome editing for Duchenne muscular dystrophy

Delivery challenges for CRISPR-Cas9 genome editing for Duchenne muscular dystrophy

#apaperaday: Toward patient-centered treatment goals for Duchenne muscular dystrophy: insights from the “Your Voice” study

Toward patient-centered treatment goals for Duchenne muscular dystrophy: insights from the “Your Voice” study

#apaperaday: Best practice recommendations for speech-language pathology in children with neuromuscular disorders: A Delphi-based consensus study

Best practice recommendations for speech-language pathology in children with neuromuscular disorders: A Delphi-based consensus study

#apaperaday: Altered muscle niche contributes to myogenic deficit in the D2-mdx model of severe DMD

Altered muscle niche contributes to myogenic deficit in the D2-mdx model of severe DMD

#apaperaday: Efficient Modulation of Exon Skipping via Antisense Circular RNAs

Efficient Modulation of Exon Skipping via Antisense Circular RNAs

#apaperaday: A Proof of Principle Proteomic Study Detects Dystrophin in Human Plasma: Implications in DMD Diagnosis and Clinical Monitoring

A Proof of Principle Proteomic Study Detects Dystrophin in Human Plasma: Implications in DMD Diagnosis and Clinical Monitoring

#apaperaday: Sources of variation in estimates of Duchenne and Becker muscular dystrophy prevalence in the United States

Sources of variation in estimates of Duchenne and Becker muscular dystrophy prevalence in the United States

#apaperaday: Dystrophin Expressing Chimeric (DEC) Cell Therapy for Duchenne Muscular Dystrophy: A First-in-Human Study with Minimum 6 Months Follow-up

Dystrophin Expressing Chimeric (DEC) Cell Therapy for Duchenne Muscular Dystrophy: A First-in-Human Study with Minimum 6 Months Follow-up

#apaperaday: Efficacy and Safety of Viltolarsen in Boys With Duchenne Muscular Dystrophy: Results From the Phase 2, Open-Label, 4-Year Extension Study

Efficacy and Safety of Viltolarsen in Boys With Duchenne Muscular Dystrophy: Results From the Phase 2, Open-Label, 4-Year Extension Study

#apaperaday: Modulating fast skeletal muscle contraction protects skeletal muscle in animal models of Duchenne muscular dystrophy

Modulating fast skeletal muscle contraction protects skeletal muscle in animal models of Duchenne muscular dystrophy

#apaperaday: A genome-wide association analysis of loss of ambulation in dystrophinopathy patients suggests multiple candidate modifiers of disease severity

A genome-wide association analysis of loss of ambulation in dystrophinopathy patients suggests multiple candidate modifiers of disease severity

#apaperaday: Unusually severe muscular dystrophy upon in-frame deletion of the dystrophin rod domain and lack of compensation by membrane-localized utrophin

Unusually severe muscular dystrophy upon in-frame deletion of the dystrophin rod domain and lack of compensation by membrane-localized utrophin

#apaperaday: Update on anti-fibrotic pharmacotherapies in skeletal muscle disease

Update on anti-fibrotic pharmacotherapies in skeletal muscle disease

#apaperaday: 3D human induced pluripotent stem cell–derived bioengineered skeletal muscles for tissue, disease and therapy modeling

3D human induced pluripotent stem cell–derived bioengineered skeletal muscles for tissue, disease and therapy modeling

#apaperaday: Six weeks of N-acetylcysteine antioxidant in drinking water decreases pathological fiber branching in MDX mouse dystrophic fast-twitch skeletal muscle

Six weeks of N-acetylcysteine antioxidant in drinking water decreases pathological fiber branching in MDX mouse dystrophic fast-twitch skeletal muscle

#apaperaday: Investigating the Impact of Delivery Routes for Exon Skipping Therapies in the CNS of DMD Mouse Models

Investigating the Impact of Delivery Routes for Exon Skipping Therapies in the CNS of DMD Mouse Models

#apaperaday: Oligonucleotide Enhancing Compound Increases Tricyclo-DNA Mediated Exon-Skipping Efficacy in the Mdx Mouse Model

Oligonucleotide Enhancing Compound Increases Tricyclo-DNA Mediated Exon-Skipping Efficacy in the Mdx Mouse Model

#apaperaday: Urine titin as a novel biomarker for Duchenne muscular dystrophy

Urine titin as a novel biomarker for Duchenne muscular dystrophy

#apaperaday: Longitudinal Assessment of Creatine Kinase, Creatine/Creatinine ratio, and Myostatin as Monitoring Biomarkers in Becker Muscular Dystrophy

Longitudinal Assessment of Creatine Kinase, Creatine/Creatinine ratio, and Myostatin as Monitoring Biomarkers in Becker Muscular Dystrophy

#apaperaday: Muscle Pathology in Dystrophic Rats and Zebrafish Is Unresponsive to Taurine Treatment, Compared to the mdx Mouse Model for Duchenne Muscular Dystrophy

Muscle Pathology in Dystrophic Rats and Zebrafish Is Unresponsive to Taurine Treatment, Compared to the mdx Mouse Model for Duchenne Muscular Dystrophy

#apaperaday: Givinostat for Becker muscular dystrophy: A randomized, placebo-controlled, double-blind study

Givinostat for Becker muscular dystrophy: A randomized, placebo-controlled, double-blind study

#apaperaday: Macrophage plasticity in Duchenne muscular dystrophy: a nexus of pathological remodelling with therapeutic implications

Macrophage plasticity in Duchenne muscular dystrophy: a nexus of pathological remodelling with therapeutic implications

#apaperaday: Variants in the Sequence of the Probe Hybridization Site May Affect MLPA Performance in Patients with Duchenne/Becker Muscular Dystrophy

Variants in the Sequence of the Probe Hybridization Site May Affect MLPA Performance in Patients with Duchenne/Becker Muscular Dystrophy

#apaperaday: Sharing "Negative" Results in Neuromuscular Research: A Positive Experience

Sharing "Negative" Results in Neuromuscular Research: A Positive Experience

Go back to all news

Sections

Results

News

#apaperaday: N-of-1 Healthcare: Challenges and Prospects for the Future of Personalized Medicine

#apaperaday: The X-linked Becker muscular dystrophy (bmx) mouse models Becker muscular dystrophy via deletion of murine dystrophin exons 45–47

#apaperaday: Microdystrophin Gene Addition Significantly Improves Muscle Functionality and Diaphragm Muscle Histopathology in a Fibrotic Mouse Model of Duchenne Muscular Dystrophy

#apaperaday: Micro-dystrophin gene therapy demonstrates long-term cardiac efficacy in a severe Duchenne muscular dystrophy model

#apaperaday: Unexpected Death of a Duchenne Muscular Dystrophy Patient in an N-of-1 Trial of rAAV9-delivered CRISPR-transactivator

#apaperaday: A Mixed-Method Study Exploring Patient-Experienced and Caregiver-Reported Benefits and Side Effects of Corticosteroid Use in Duchenne Muscular Dystrophy

#apaperaday: Delivery challenges for CRISPR-Cas9 genome editing for Duchenne muscular dystrophy

#apaperaday: Toward patient-centered treatment goals for Duchenne muscular dystrophy: insights from the “Your Voice” study

#apaperaday: Best practice recommendations for speech-language pathology in children with neuromuscular disorders: A Delphi-based consensus study

#apaperaday: Altered muscle niche contributes to myogenic deficit in the D2-mdx model of severe DMD

#apaperaday: Efficient Modulation of Exon Skipping via Antisense Circular RNAs

#apaperaday: A Proof of Principle Proteomic Study Detects Dystrophin in Human Plasma: Implications in DMD Diagnosis and Clinical Monitoring

#apaperaday: Sources of variation in estimates of Duchenne and Becker muscular dystrophy prevalence in the United States

#apaperaday: Dystrophin Expressing Chimeric (DEC) Cell Therapy for Duchenne Muscular Dystrophy: A First-in-Human Study with Minimum 6 Months Follow-up

#apaperaday: Efficacy and Safety of Viltolarsen in Boys With Duchenne Muscular Dystrophy: Results From the Phase 2, Open-Label, 4-Year Extension Study

#apaperaday: Modulating fast skeletal muscle contraction protects skeletal muscle in animal models of Duchenne muscular dystrophy

#apaperaday: A genome-wide association analysis of loss of ambulation in dystrophinopathy patients suggests multiple candidate modifiers of disease severity

#apaperaday: Unusually severe muscular dystrophy upon in-frame deletion of the dystrophin rod domain and lack of compensation by membrane-localized utrophin

#apaperaday: Update on anti-fibrotic pharmacotherapies in skeletal muscle disease

#apaperaday: 3D human induced pluripotent stem cell–derived bioengineered skeletal muscles for tissue, disease and therapy modeling

#apaperaday: Six weeks of N-acetylcysteine antioxidant in drinking water decreases pathological fiber branching in MDX mouse dystrophic fast-twitch skeletal muscle

#apaperaday: Investigating the Impact of Delivery Routes for Exon Skipping Therapies in the CNS of DMD Mouse Models

#apaperaday: Oligonucleotide Enhancing Compound Increases Tricyclo-DNA Mediated Exon-Skipping Efficacy in the Mdx Mouse Model

#apaperaday: Urine titin as a novel biomarker for Duchenne muscular dystrophy

#apaperaday: Longitudinal Assessment of Creatine Kinase, Creatine/Creatinine ratio, and Myostatin as Monitoring Biomarkers in Becker Muscular Dystrophy

#apaperaday: Muscle Pathology in Dystrophic Rats and Zebrafish Is Unresponsive to Taurine Treatment, Compared to the mdx Mouse Model for Duchenne Muscular Dystrophy

#apaperaday: Givinostat for Becker muscular dystrophy: A randomized, placebo-controlled, double-blind study

#apaperaday: Macrophage plasticity in Duchenne muscular dystrophy: a nexus of pathological remodelling with therapeutic implications

#apaperaday: Variants in the Sequence of the Probe Hybridization Site May Affect MLPA Performance in Patients with Duchenne/Becker Muscular Dystrophy

#apaperaday: Sharing "Negative" Results in Neuromuscular Research: A Positive Experience

DMD Emergency Program Provides Aid to Duchenne Families in Ukraine

Register for Duchenne Care Conference 2023

ADC open to accept applications for Duchenne Centers Accreditation