Casimersen (AMONDYS 45™) is a therapeutic antisense oligonucleotide designed for patients with Duchenne Muscular Dystrophy (DMD) whose gene mutation is amenable to exon 45 skipping. DMD is a genetic disorder characterized by the absence of dystrophin, a crucial protein for muscle function. This results in progressive muscle degeneration and loss of mobility, eventually affecting the heart and lungs. Casimersen works by binding to the mutated pre-mRNA, skipping exon 45 to …

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1. Propensity Score Matching      A statistical method used to balance groups in observational studies, reducing bias by matching individuals with similar characteristics. This helps improve the validity of comparisons, especially in studies where randomization isn't possible. 2. North Star Ambulatory Assessment (NSAA) and North Star Assessment    These are clinical tools used to measure motor abilities in patients with neuromuscular disorders. The NSAA, for example, is frequently used …

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Myotonic Dystrophy (DM) is a genetic disorder that affects muscle function and other systems of the body. There are two main types: 1. Myotonic Dystrophy Type 1 (DM1): Also known as Steinert's disease, DM1 is caused by a mutation in the "DMPK gene" (Dystrophia Myotonica Protein Kinase). The mutation involves an expansion of CTG trinucleotide repeats in this gene, leading to symptoms such as prolonged muscle contractions (myotonia), muscle weakness, …

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