#apaperaday: Burden of Disease of Duchenne Muscular Dystrophy in Denmark
In today’s #apaperaday, Prof. Aartsma-Rus reads and comments on the paper titled: Burden of Disease of Duchenne Muscular Dystrophy in…
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#apaperaday: Draft Guidance for Industry Duchenne Muscular Dystrophy, Becker Muscular Dystrophy, and Related Dystrophinopathies - Developing Potential Treatments for the Entire Spectrum of Disease
In today’s #apaperaday, Prof. Aartsma-Rus reads and comments on the paper titled: Draft Guidance for Industry Duchenne Muscular Dystrophy, Becker…
#apaperaday: Diverse Cardiac Phenotype of Becker Muscular Dystrophy: Under-Recognized Subclinical Cardiomyopathy Due to Partial Dystrophin Deficiency in a Contemporary Era
In today’s #apaperaday, Prof. Aartsma-Rus reads and comments on the paper titled: Diverse Cardiac Phenotype of Becker Muscular Dystrophy: Under-Recognized…
#apaperaday: Use of plasmapheresis to lower anti-AAV antibodies in nonhuman primates with pre-existing immunity to AAVrh74
In today’s #apaperaday, Prof. Aartsma-Rus reads and comments on the paper titled: Use of plasmapheresis to lower anti-AAV antibodies in…
#apaperaday: Efficacy and Safety of Vamorolone Over 48 Weeks in Boys With Duchenne Muscular Dystrophy
In today’s #apaperaday, Prof. Aartsma-Rus reads and comments on the paper titled: Efficacy and Safety of Vamorolone Over 48 Weeks…
#apaperaday: Different bone health progression patterns and early-stage risk marker in glucocorticoid-treated ambulatory Duchenne muscular dystrophy
In today’s #apaperaday, Prof. Aartsma-Rus reads and comments on the paper titled: Different bone health progression patterns and early-stage risk…
#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
