With the ability to provide non-invasive, three dimensional, longitudinal, and quantitative data—PET, SPECT, and fluorescence imaging (FMT) enable translational imaging of: Biodistribution Targeting Kinetics Clearance Pathways
Making Sense of Antisense Oligonucleotide-Based Therapies in Muscular Dystrophies
Good news for the Duchenne Muscular Dystrophy (DMD) community. On June 8th, BioMarin announced the filing of a Marketing Authorization Application to the European Medicines Agency for Drisapersen, an antisense-mediated exon 51-skipping compound able to target the most prevalent genetic mutations responsible for the lack of production of functional dystrophin. …