Paper a day Archives • World Duchenne

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#apaperaday: Targeted Antisense Oligonucleotide-Mediated Skipping of Murine Postn Exon 17 Partially Addresses Fibrosis in D2.mdx Mice

In today’s #apaperaday, Prof. Aartsma-Rus reads and comments on the paper titled: Targeted Antisense Oligonucleotide-Mediated Skipping of Murine Postn Exon…

#apaperaday: Tamoxifen may contribute to preserve cardiac function in Duchenne muscular dystrophy

In today’s #apaperaday, Prof. Aartsma-Rus reads and comments on the paper titled: Tamoxifen may contribute to preserve cardiac function in…

#apaperaday: Eteplirsen Treatment for Duchenne Muscular Dystrophy: A Qualitative Patient Experience Study

In today’s #apaperaday, Prof. Aartsma-Rus reads and comments on the paper titled: Eteplirsen Treatment for Duchenne Muscular Dystrophy: A Qualitative…

#apaperaday: Givinostat: First Approval

In today’s #apaperaday, Prof. Aartsma-Rus reads and comments on the paper titled: Givinostat: First Approval Today's pick is from the…

#apaperaday: How to pay for individualized genetic medicines

In today’s #apaperaday, Prof. Aartsma-Rus reads and comments on the paper titled: How to pay for individualized genetic medicines Paper…

#apaperaday: Current Classification of Canine Muscular Dystrophies and Identification of New Variants

In today’s #apaperaday, Prof. Aartsma-Rus reads and comments on the paper titled: Current Classification of Canine Muscular Dystrophies and Identification…

#apaperaday:Patient- and caregiver-reported impact of symptoms in Duchenne muscular dystrophy

In today’s #apaperaday, Prof. Aartsma-Rus reads and comments on the paper titled: Patient- and caregiver-reported impact of symptoms in Duchenne…

#apaperaday: Potential limitations of microdystrophin gene therapy for Duchenne muscular dystrophy

In today’s #apaperaday, Prof. Aartsma-Rus reads and comments on the paper titled: Potential limitations of microdystrophin gene therapy for Duchenne…

#apaperaday: Perspectives on and Experiences With Bullying From Youth With Neuromuscular Conditions

In today’s #apaperaday, Prof. Aartsma-Rus reads and comments on the paper titled: Perspectives on and Experiences With Bullying From Youth…

#apaperaday: Immune-mediated myositis following gene therapy for Duchenne muscular dystrophy: a case report

In today’s #apaperaday, Prof. Aartsma-Rus reads and comments on the paper titled: Immune-mediated myositis following gene therapy for Duchenne muscular…

#apaperaday: Mitochondria and Reactive Oxygen Species: The Therapeutic Balance of Powers for Duchenne Muscular Dystrophy

In today’s #apaperaday, Prof. Aartsma-Rus reads and comments on the paper titled: Mitochondria and Reactive Oxygen Species: The Therapeutic Balance…

#apaperaday: Transcriptomic gene signatures measure satellite cell activity in muscular dystrophies

In today’s #apaperaday, Prof. Aartsma-Rus reads and comments on the paper titled: Transcriptomic gene signatures measure satellite cell activity in…

#apaperaday: Severe gastrointestinal problems in Duchenne muscular dystrophy: A case series

In today’s #apaperaday, Prof. Aartsma-Rus reads and comments on the paper titled: Severe gastrointestinal problems in Duchenne muscular dystrophy: A…

#apaperaday: Chronic N-acetyl cysteine treatment does not improve respiratory system performance in the mdx mouse model of Duchenne muscular dystrophy

In today’s #apaperaday, Prof. Aartsma-Rus reads and comments on the paper titled: Chronic N-acetyl cysteine treatment does not improve respiratory…

#apaperaday: Functional trajectories before and after loss of ambulation in Duchenne muscular dystrophy and implications for clinical trials

In today’s #apaperaday, Prof. Aartsma-Rus reads and comments on the paper titled: Functional trajectories before and after loss of ambulation…

#apaperaday: Newborn Screening for Duchenne Muscular Dystrophy – The Time is Now

In today’s #apaperaday, Prof. Aartsma-Rus reads and comments on the paper titled: Newborn Screening for Duchenne Muscular Dystrophy – The…

#apaperaday: Stride Velocity 95th Centile Detects Decline in Ambulatory Function Over Shorter Intervals than the 6-Minute Walk Test or North Star Ambulatory Assessment in Duchenne Muscular Dystrophy

In today’s #apaperaday, Prof. Aartsma-Rus reads and comments on the paper titled: Stride Velocity 95th Centile Detects Decline in Ambulatory…

#apaperaday: Social difficulties and care burden of adult Duchenne muscular dystrophy in Japan: a questionnaire survey based on the Japanese Registry of Muscular Dystrophy (Remudy)

In today’s #apaperaday, Prof. Aartsma-Rus reads and comments on the paper titled: Social difficulties and care burden of adult Duchenne…

#apaperaday: Chimeric Cell Therapies as a Novel Approach for Duchenne Muscular Dystrophy (DMD) and Muscle Regeneration

In today’s #apaperaday, Prof. Aartsma-Rus reads and comments on the paper titled: Chimeric Cell Therapies as a Novel Approach for…

#apaperaday: CRISPR-Based Gene Therapies: From Preclinical to Clinical Treatments

In today’s #apaperaday, Prof. Aartsma-Rus reads and comments on the paper titled: CRISPR-Based Gene Therapies: From Preclinical to Clinical Treatments…

#apaperaday: Barriers to diverse clinical trial participation in Duchenne muscular dystrophy: Engaging Hispanic/Latina caregivers and health professionals

In today’s #apaperaday, Prof. Aartsma-Rus reads and comments on the paper titled: Barriers to diverse clinical trial participation in Duchenne…

#apaperaday: Integrated genomic, proteomic and cognitive assessment in Duchenne Muscular Dystrophy suggest astrocyte centric pathology

In today’s #apaperaday, Prof. Aartsma-Rus reads and comments on the paper titled: Integrated genomic, proteomic and cognitive assessment in Duchenne…

#apaperaday: Development and Pilot Validation of the DuMAND Checklist to Screen for Duchenne Muscular Dystrophy-Associated Neurobehavioral Difficulties (DuMAND)

In today’s #apaperaday, Prof. Aartsma-Rus reads and comments on the paper titled: Development and Pilot Validation of the DuMAND Checklist…

#apaperaday: A rare complex structural variant of novel intragenic inversion combined with reciprocal translocation t(X;1)(p21.2;p13.3) in Duchenne muscular dystrophy

In today’s #apaperaday, Prof. Aartsma-Rus reads and comments on the paper titled: A rare complex structural variant of novel intragenic…

#apaperaday: Respiratory comorbidities and treatments in Duchenne muscular dystrophy: impact on life expectancy and causes of death

In today’s #apaperaday, Prof. Aartsma-Rus reads and comments on the paper titled: Respiratory comorbidities and treatments in Duchenne muscular dystrophy:…

#apaperaday: Discordant Monozygotic Triplets with Duchenne Muscular Dystrophy

In today’s #apaperaday, Prof. Aartsma-Rus reads and comments on the paper titled: Discordant Monozygotic Triplets with Duchenne Muscular Dystrophy The…

#apaperaday: Predictors of Loss of Ambulation in Duchenne Muscular Dystrophy: A Systematic Review and Meta-Analysis

In today’s #apaperaday, Prof. Aartsma-Rus reads and comments on the paper titled: Predictors of Loss of Ambulation in Duchenne Muscular…

#apaperaday: Assessing the Benefits and Harms Associated with Early Diagnosis from the Perspective of Parents with Multiple Children Diagnosed with Duchenne Muscular Dystrophy

In today’s #apaperaday, Prof. Aartsma-Rus reads and comments on the paper titled: Assessing the Benefits and Harms Associated with Early…

#apaperaday: Genetic counseling for the dystrophinopathies—Practice resource of the National Society of Genetic Counselors

In today’s #apaperaday, Prof. Aartsma-Rus reads and comments on the paper titled: Genetic counseling for the dystrophinopathies—Practice resource of the…

#apaperaday: Long-term clinical follow-up of a family with Becker muscular dystrophy associated with a large deletion in the DMD gene

In today’s #apaperaday, Prof. Aartsma-Rus reads and comments on the paper titled: Long-term clinical follow-up of a family with Becker…

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#apaperaday: Targeted Antisense Oligonucleotide-Mediated Skipping of Murine Postn Exon 17 Partially Addresses Fibrosis in D2.mdx Mice

#apaperaday: Tamoxifen may contribute to preserve cardiac function in Duchenne muscular dystrophy

#apaperaday: Eteplirsen Treatment for Duchenne Muscular Dystrophy: A Qualitative Patient Experience Study

#apaperaday: Givinostat: First Approval

#apaperaday: How to pay for individualized genetic medicines

#apaperaday: Current Classification of Canine Muscular Dystrophies and Identification of New Variants

#apaperaday:Patient- and caregiver-reported impact of symptoms in Duchenne muscular dystrophy

#apaperaday: Potential limitations of microdystrophin gene therapy for Duchenne muscular dystrophy

#apaperaday: Perspectives on and Experiences With Bullying From Youth With Neuromuscular Conditions

#apaperaday: Immune-mediated myositis following gene therapy for Duchenne muscular dystrophy: a case report

#apaperaday: Mitochondria and Reactive Oxygen Species: The Therapeutic Balance of Powers for Duchenne Muscular Dystrophy

#apaperaday: Transcriptomic gene signatures measure satellite cell activity in muscular dystrophies

#apaperaday: Severe gastrointestinal problems in Duchenne muscular dystrophy: A case series

#apaperaday: Chronic N-acetyl cysteine treatment does not improve respiratory system performance in the mdx mouse model of Duchenne muscular dystrophy

#apaperaday: Functional trajectories before and after loss of ambulation in Duchenne muscular dystrophy and implications for clinical trials

#apaperaday: Newborn Screening for Duchenne Muscular Dystrophy – The Time is Now

#apaperaday: Stride Velocity 95th Centile Detects Decline in Ambulatory Function Over Shorter Intervals than the 6-Minute Walk Test or North Star Ambulatory Assessment in Duchenne Muscular Dystrophy

#apaperaday: Social difficulties and care burden of adult Duchenne muscular dystrophy in Japan: a questionnaire survey based on the Japanese Registry of Muscular Dystrophy (Remudy)

#apaperaday: Chimeric Cell Therapies as a Novel Approach for Duchenne Muscular Dystrophy (DMD) and Muscle Regeneration

#apaperaday: CRISPR-Based Gene Therapies: From Preclinical to Clinical Treatments

#apaperaday: Barriers to diverse clinical trial participation in Duchenne muscular dystrophy: Engaging Hispanic/Latina caregivers and health professionals

#apaperaday: Integrated genomic, proteomic and cognitive assessment in Duchenne Muscular Dystrophy suggest astrocyte centric pathology

#apaperaday: Development and Pilot Validation of the DuMAND Checklist to Screen for Duchenne Muscular Dystrophy-Associated Neurobehavioral Difficulties (DuMAND)

#apaperaday: A rare complex structural variant of novel intragenic inversion combined with reciprocal translocation t(X;1)(p21.2;p13.3) in Duchenne muscular dystrophy

#apaperaday: Respiratory comorbidities and treatments in Duchenne muscular dystrophy: impact on life expectancy and causes of death

#apaperaday: Discordant Monozygotic Triplets with Duchenne Muscular Dystrophy

#apaperaday: Predictors of Loss of Ambulation in Duchenne Muscular Dystrophy: A Systematic Review and Meta-Analysis

#apaperaday: Assessing the Benefits and Harms Associated with Early Diagnosis from the Perspective of Parents with Multiple Children Diagnosed with Duchenne Muscular Dystrophy

#apaperaday: Genetic counseling for the dystrophinopathies—Practice resource of the National Society of Genetic Counselors

#apaperaday: Long-term clinical follow-up of a family with Becker muscular dystrophy associated with a large deletion in the DMD gene

Road to independence: Documentary about young people with NMD

Webinar Recording: Understanding the role of dystrophin in the brain in DMD/BMD

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