ABSTRACT Background Duchenne muscular dystrophy (DMD) is the most common and severe form of muscular dystrophy, primarily affecting skeletal muscle and leading to premature death. Although the loss of dystrophin has long been recognised as the primary cause of the disease, no definitive cure is currently available. As a consequence, therapeutic efforts
Raffaele Epis +5 more
wiley +1 more source
RRAEDy: adaptive latent linearization of nonlinear dynamical systems. [PDF]
Mounayer J +4 more
europepmc +1 more source
Supercritical Angle Fluorescence and Raman Techniques in Biophotonics
Supercritical angle fluorescence (SAF) microscopy and supercritical angle Raman (SAR) spectroscopy have emerged as powerful tools in the field of biophotonics, enabling high‐resolution imaging and spectroscopy of biological specimens near interfaces. This review article provides a comprehensive overview of the principles, techniques, applications, and ...
Subir Das +2 more
wiley +1 more source
A novel spatiotemporal decomposition and identification of sparse equations for human brain deformation. [PDF]
Arani AHG +4 more
europepmc +1 more source
The Economic Burden of Duchenne Muscular Dystrophy: A Systematic Review. [PDF]
Landfeldt E +6 more
europepmc +1 more source
ABSTRACT Aims We report 5‐year results from a phase 1/2a study of delandistrogene moxeparvovec, a recombinant adeno‐associated virus serotype rh74 vector‐based gene therapy for Duchenne muscular dystrophy (DMD), with post hoc analyses contextualizing functional outcomes. Methods Four ambulatory patients with DMD (≥ 4–< 8 years at enrollment) entered an
Jerry R. Mendell +10 more
wiley +1 more source
Impact of C4BPA on Muscle progenitor cell differentiation: insights for Duchenne muscular dystrophy treatment. [PDF]
Fernández-Simón E +12 more
europepmc +1 more source
Emerging roles of microRNAs and other non-coding transcriptome in muscular dystrophies. [PDF]
Abdelrehim FG +5 more
europepmc +1 more source
Creatine/Creatinine Ratio and Myostatin as Biomarkers to Monitor Muscle Function in Duchenne Muscular Dystrophy Patients. [PDF]
Degan C +7 more
europepmc +1 more source
RNA Therapeutics for Duchenne Muscular Dystrophy: Exon Skipping, RNA Editing, and Translational Insights from Genome-Edited Microminipig Models. [PDF]
Chassin A +4 more
europepmc +1 more source

