Available Forms: Injection
Available Strengths: 50 mg/mL
Reference Brands: Exondys 51 (USA/EU)
Category: Neurology
Eteplirsen is an antisense oligonucleotide used to treat Duchenne Muscular Dystrophy (DMD) in patients with a confirmed mutation amenable to exon 51 skipping. It works by promoting the production of functional dystrophin protein, which is crucial for maintaining muscle strength and function. Eteplirsen is administered as an intravenous infusion, typically once a week.
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Eteplirsen is used to treat Duchenne muscular dystrophy (DMD) in patients who have a confirmed mutation of the DMD gene that is amenable to exon 51 skipping.
Eteplirsen is made from a synthetic phosphorodiamidate morpholino oligomer (PMO) that binds to specific RNA sequences to promote exon 51 skipping during dystrophin protein synthesis.
The trade name of Eteplirsen is Exondys 51.
Eteplirsen is made by Sarepta Therapeutics, Inc.
The generic name of the product is Eteplirsen.
The brand name of Eteplirsen is Exondys 51.
Eteplirsen is manufactured by Sarepta Therapeutics, Inc. in the United States.
Strength: 125 mg/mL, 200 mg, 250 mg, 300 mg, 500 mg, 100 mg/mL
Form: Tablet / Capsule/ Injection / Oral syrup
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