Casimersen Suppliers & Bulk Manufacturers

Available Forms: Injection

Available Strengths: 50 mg/ml

Reference Brands: Amondys 45 (USA)

Category: Neurology

Product Description:

Casimersen, marketed under the brand name Amondys 45, is an antisense oligonucleotide medication specifically designed for the treatment of Duchenne muscular dystrophy (DMD) in patients with a confirmed mutation of the dystrophin gene that is amenable to exon 45 skipping. It is a phosphorodiamidate morpholino oligomer (PMO) that targets dystrophin pre-mRNA, enabling the cellular machinery to skip the defective exon and produce a shortened but functional dystrophin protein.

Duchenne muscular dystrophy is a rare, progressive genetic disorder that primarily affects boys and is characterized by the absence or deficiency of dystrophin, a critical protein for muscle integrity and function. The lack of dystrophin leads to progressive muscle weakness, loss of mobility, and reduced life expectancy. Casimersen offers a targeted therapeutic approach by addressing the underlying genetic defect, aiming to slow disease progression and improve muscle function. The medication is administered via intravenous infusion under strict medical supervision, with treatment tailored to the patient’s specific genetic mutation and clinical condition.