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Soft genome editing based on CRISPR nickases
Orthogonal proteomics methods warrant the development of Duchenne muscular dystrophy biomarkers
Networking to optimize Dmd exon 53 skipping in the brain of mdx52 mouse model
Lessons learned from the first national population-based genetic carrier-screening program for Duchenne muscular dystrophy
Shepherding precision gene editing with CRISPR-Cas9 variants and adenoviral vectors
Next generation exon 51 skipping antisense oligonucleotides for Duchenne muscular dystrophy
Outcome measures in Duchenne muscular dystrophy
T cell responses to dystrophin in a natural history study of Duchenne muscular dystrophy
Next steps for the optimization of exon therapy for Duchenne muscular dystrophy
CRISPR applications for Duchenne muscular dystrophy
Real-world and natural history data for drug evaluation in Duchenne muscular dystrophy
BMI-z scores of boys with Duchenne muscular dystrophy already begin to increase before losing ambulation
Effects of muscle damage on P-31-MRS indices of energetic status and sarcolemma integrity in young mdx mice
Genotype-related respiratory progression in Duchenne muscular dystrophy
Occurrence of symptoms in different stages of Duchenne muscular dystrophy and their impact on social participation
Penalized regression calibration
Prevalence of bladder and bowel dysfunction in Duchenne muscular dystrophy using the Childhood Bladder and Bowel Dysfunction Questionnaire
Preserved thenar muscles in non-ambulant Duchenne muscular dystrophy patients
Peripheral blood transcriptome profiling enables monitoring disease progression in dystrophic mice and patients
Resting-state functional MRI shows altered default-mode network functional connectivity in Duchenne muscular dystrophy patients
Clinical management of Duchenne muscular dystrophy in the Netherlands
How patient organizations can drive FAIR data efforts to facilitate research and health care
Compliance to DMD care considerations in the Netherlands
Novel free-circulating and extracellular vesicle-derived miRNAs dysregulated in Duchenne muscular dystrophy
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